Archive for the ‘Gene Therapy Research’ Category

The Global Oncolytic Virus Immunotherapy Market Will Be Driven By Increasing Number Of Clinical Trials Along With New Product Launches Over Next 5 Years Says Kuick Research

NEW DELHI, Oct. 8, 2020 /PRNewswire/ -- "Global Oncolytic Virus Immunotherapy Market, Dosage, Price & Clinical Trials Outlook 2026" Report Highlights:

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https://www.kuickresearch.com/report-oncolytic-virus-immunotherapy-market-size-sales-clinical-trials-cancer-oncology-melanoma-report-talimogene-laherparepvec-imlygic--oncorine

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Increase in prevalence and mortality rate associated with cancer have always remained a challenge to the modern medicine with respect to the development of more reliable therapies. In the past few years, one of the greatest breakthrough novel cancer therapy that has been developed by the researchers is the oncolytic virus immunotherapy. The overall procedure of the therapy is based on the ability of the oncolytic virus to infect and further lyse the tumor cells by boosting the immune system of the patient. Some of the virus strains that have been tested and proved to be adding to the various applications of the therapy are canine distemper virus, adenovirus, vaccinia virus and reovirus in the preclinical and clinical studies.

The viral toxicity and the in-vitro manipulation of the virus is estimated to be providing successful clinical outcomes, leading to the enhancement and the expansion of the market linked with the therapy. All the clinical trials associated with the therapy are underway but all are estimated to look forward by bringing numerous opportunities for the cancer patients. The market concerned with the growth of the oncolytic virus therapy is focused on increasing the research and development sector, which is apparently considered as an important asset for the therapy in order to change the stringent cancer treatment paradigm available in the industry for a long period of time. The current clinical platform is estimated to be developing a competitive landscape for the other cancer markets as the therapy of interest is getting fragmented at a high speed for different cancer types.

The blockbuster model held by the therapy is also believed to be driving the large shareholder value of the market in the present as well as in the upcoming years. The increase in the perception of highly expensive drugs available under cell and gene therapy are expected to be one of the most regulated drivers for the global expansion of the oncolytic therapy. As a result of strong clinical background and healthy research and development sector, oncolytic virus therapy is becoming more prominent in the cancer landscape. With the primary goal of revolutionizing the cancer therapeutics market, it is estimated that the therapy in a short period of time will be successful in re-defining the unstructured cancer paradigm for the patients with advanced cancers.

Being not only restricted to clinical benefits, the therapy is moving forward to provide better post-treatment lives to the cancer patients. The therapy in the clinical studies have been able to put forward the value of the patients in terms of clinical outcomes as well as patient-related outcome measures, thus causing a wider acceptance to the therapy in the cancer therapeutics market. Some of the primary aim associated with the current clinical pipeline of the therapy involves limited cost expansion and increased success rate. Prominent factors like these are estimated to enhance access of the patient population to effective oncolytic virus therapy.

The future view of the oncolytic virus therapy is estimated to be enabling such innovative mechanisms that would be responsible for providing tremendous amount of opportunities to the cancer patients. Different gene-editing technologies, advancement in genomics and many others will map the therapy among the best-cancer regimens ever discovered. The leverage provided to the therapy in terms of opportunities depicts the possibility of the therapy to get recognized as a real cure to the cancer patients and eventually a dominating market. With the advent of the therapy for the cancer platform, there has been a significant transformation in the current cancer therapeutics market. In financial services, the market is estimated to perform with accuracy and speed in the future years.

As per " Global Oncolytic Virus Immunotherapy Market, Dosage, Price & Clinical Trials Outlook 2026" report findings, it is estimated that the total inflation of the market with respect to clinical platform, wider acceptance in a short period of time will drive the market in the upcoming years. Gradual increase in the interest of the researchers towards oncolytic virus therapy will develop the market which will eventually bring complete revolution in terms of applications to the cancer patients. The future market insight for the therapy is analyzed to provide unlimited number of trends and opportunities, leading to aggregation of all the cancer related bio-pharmaceutical companies and research centers towards the further development and commercialization of the therapy.

Contact:

Neeraj Chawla[emailprotected]+91-9810410366

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SOURCE Kuick Research

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Global Oncolytic Virus Immunotherapy Market To Reach US$ 750 Million By 2026 With 500% Absolute Growth - PR Newswire India

The Food and Drug Administration (FDA) has granted Fast Track designation to the investigational gene therapy BMN 307 (BioMarin Pharmaceutical) for the treatment of patients with phenylketonuria (PKU).

BMN 307 is an adeno-associated virus type 5 (AAV5)-phenylalanine hydroxylase (PAH) gene therapy designed to normalize blood phenylalanine (Phe) concentration levels in patients with PKU by inserting a correct copy of the PAH gene into liver cells.

The Company recently initiated an open-label phase 1/2 study (PHEARLESS) to evaluate the safety, efficacy and tolerability of a single intravenous administration of BMN 307 in adults with PKU with PAH deficiency. The study will assess whether a single dose of treatment can restore natural Phe metabolism, increase plasma Phe levels, and enable a normalization of diet in patients with PKU.

Fast Track designation combined with our ability to conduct our clinical studies incorporating material manufactured using a commercial-ready process will further facilitate rapid clinical development of BMN 307 gene therapy, said Hank Fuchs, MD, President, Worldwide Research and Development at BioMarin. We are looking forward to working closely with the FDA, as well as other health agencies, to evaluate the safety and efficacy of this promising investigational gene therapy as we continue our unwavering 15-year commitment to advance the standard of care for people with PKU.

The FDAs Fast Track designation allows for expedited review of therapies that are meant to treat serious or life-threatening conditions. Generally, the designation is granted to drugs that are expected to have an impact on factors such as survival and daily functioning.

For more information visit biomarin.com.

BioMarin, pioneer in phenylketonuria (PKU) and gene therapy, receives FDA Fast Track designation for PKU investigational gene therapy, BMN 307. https://www.prnewswire.com/news-releases/biomarin-pioneer-in-phenylketonuria-pku-and-gene-therapy-receives-fda-fast-track-designation-for-pku-investigational-gene-therapy-bmn-307-301144678.html. Accessed October 5, 2020.

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Investigational Gene Therapy for Phenylketonuria Gets Fast Tracked - Monthly Prescribing Reference

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Establishment of Social Cooperation Program "Protein Degradation Drug Discovery"

TOKYO, Japan I October 8, 2020 I The University of Tokyo and Eisai Co., Ltd. announced today a collaboration aiming for the development and drug discovery of targeted protein degradation technology has been created, with the establishment of a social cooperation program, "Protein Degradation Drug Discovery". The research time span will last five years from October 1, 2020 to September 30, 2025.

The social cooperation program is established and operated based on funds of private organizations dedicated to conducting research in collaboration with the University of Tokyo regarding shared issues of high common concern.

The "Protein Degradation Drug Discovery" course is to be established within the Graduate School of Pharmaceutical Sciences, the University of Tokyo. Dr. Mikihiko Naito, Former Director of the Division of Molecular Target and Gene Therapy Products, National Institute of Health Sciences, has been inaugurated as a project professor for this program and will lead research with protein degradation technology including SNIPER. This research will combine the world's most advanced ubiquitin-proteasome research as conducted in the graduate school with drug discovery knowledge fostered by Eisai, for the development of new protein degradation technology towards proteins targeted by drugs and the promotion of drug discovery research based on this technology. In addition, through this research, the course will educate and train the next generation of leaders in this research field.

Targeted protein degradation is a series of technologies in which precisely designed compounds force target proteins into proximity with E3 ubiquitin ligase and apply the ubiquitin-proteasome system to induce degradation of the target proteins. The technology provides a means of creating medicines for not only conventional targets such as specific enzymes and receptors, but also disease-related proteins for which drug discovery up to this point has been difficult. Through the development of this technology and drug discovery, the University of Tokyo and Eisai aim to provide new treatment options to patients for which treatment options were previously limited.

About SNIPER

SNIPER (Specific and Nongenetic IAP-dependent Protein Eraser) is a compound which utilizes the ubiquitin-proteasome system to degrade target proteins. This compound is a "hybrid compound", and consists of a moiety that binds to the target protein and a moiety that binds to E3 ubiquitin ligase (IAPs) with an appropriate linker. Designing this compound requires advanced medicinal chemistry and cutting-edge structural biology. When the SNIPER compound brings the target protein and E3 ubiquitin ligase (IAPs) into proximity (Step 1 in the chart below), ubiquitin as a protein degradation tag transfers from the E2 ubiquitin conjugating enzyme to the target protein (2) for recognition of the protein by the proteasome and subsequent degradation (3).

Conventional small-molecule inhibitors bind to the active moiety of target enzymes, and express pharmacological activity by inhibiting the activity thereof. On the other hand, because SNIPER exhibits pharmacological properties by target protein degradation as described above, it is not only expected to exhibit different pharmacological activity from small-molecule inhibitors; it is also predicted to target proteins that do not have enzymatic activity. Similar technologies include PROTAC (PROteolysis TArgeting Chimeras) and Degronimid.

About the Ubiquitin-Proteasome System

The ubiquitin-proteasome system is one of the naturally occurring mechanisms for controlling the degradation of unneeded proteins and is in control of critical movements relating to the preservation of life including cell cycle, transcription regulation, and signal transmission. When ubiquitin as a protein degradation tag connects to unneeded proteins through agents such as the E3 ubiquitin ligase, it marks the protein for recognition by the proteasome for subsequent degradation. In recent years, the relation between the ubiquitin-proteasome system and major human diseases including cancer and neurodegeneration is becoming evident.

About Eisai

Eisai Co., Ltd. is a leading global research and development-based pharmaceutical company headquartered in Japan. We define our corporate mission as "giving first thought to patients and their families and to increasing the benefits health care provides," which we call our human health care (hhc) philosophy. With approximately 10,000 employees working across our global network of R&D facilities, manufacturing sites and marketing subsidiaries, we strive to realize our hhc philosophy by delivering innovative products to address unmet medical needs, with a particular focus in our strategic areas of Neurology and Oncology. As a global pharmaceutical company, our mission extends to patients around the world through our investment and participation in partnership-based initiatives to improve access to medicines in developing and emerging countries.

For more information about Eisai Co., Ltd., please visit https://www.eisai.com

SOURCE: Eisai

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The University of Tokyo and Eisai Announce Research Collaboration for the Development and Drug Discovery of Targeted Protein Degradation Technology |...

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Cell and Gene Therapy Consumables Market Excessive Growth Opportunities and Trends Estimated in Report Forecast Forecast, 2020-2026 - re:Jerusalem

New York, Oct. 08, 2020 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Regenerative Medicine Market by Product, Application, Geography - Global Forecast to 2025" - https://www.reportlinker.com/p04700208/?utm_source=GNW However, the high cost of cell and gene therapies and ethical concerns related to the use of embryonic stem cells in research and development are expected to restrain the growth of this market during the forecast period.The cell therapies segment accounted for the highest growth rate in the regenerative medicine market, by product, during the forecast periodBased on products, the regenerative medicine market is segmented into tissue-engineered products, cell therapies, gene therapies, and progenitor and stem cell therapies.The cell therapies segment accounted for the highest growth rate in the regenerative medicine market in 2019.

The increasing adoption of tissue-engineered products for the treatment of chronic wounds and musculoskeletal disorders and the rising funding for the R&D of regenerative medicine products and therapies are the major factors driving the growth of this segment.

Oncology segment accounted for highest CAGRBased on applications, the regenerative medicine market is segmented into musculoskeletal disorders, wound care, oncology, ocular disorders, dental, and other applications.In 2019, the oncology segment accounted for the highest growth rate.

This can be attributed to the rising prevalence of orthopedic diseases, growing geriatric population, increasing number of stem cell research projects, growing number of clinical researches/trials, and the rich pipeline of stem cell products for the treatment of musculoskeletal disorders.

Europe: The fastest-growing region regenerative medicine marketThe global regenerative medicine market is segmented into North America, Europe, the Asia Pacific, and Rest of the World.The North America region is projected to grow at the highest CAGR during the forecast period in 2019.

The growth in the North American regenerative medicine market can be attributed to rising stem cell banking, tissue engineering, and drug discovery in the region; expansion of the healthcare sector; and the high adoption of stem cell therapy and cell immunotherapies for the treatment of cancer and chronic diseases.

The primary interviews conducted for this report can be categorized as follows: By Company Type: Tier 1 - 20%, Tier 2 - 45%, and Tier 3 - 35% By Designation: C-level - 30%, D-level - 20%, and Others - 50% By Region: North America - 36%, Europe - 25%, Asia Pacific - 27%, and Rest of the World 12%

Lits of companies Profiled in the Report: 3M (US) Allergan plc (Ireland) Amgen, Inc. (US) Aspect Biosystems (Canada) bluebird bio (US) Kite Pharma (US) Integra LifeSciences Holdings Corporation (US) MEDIPOST Co., Ltd. (South Korea) Medtronic plc (Ireland) Anterogen Co., Ltd. (South Korea) MiMedx Group (US) Misonix (US) Novartis AG (Switzerland) Organogenesis Inc. (US) Orthocell Limited (Australia) Corestem, Inc. (South Korea) Spark Therapeutics (US) APAC Biotech (India) Shenzhen Sibiono GeneTech Co., Ltd. (China) Smith & Nephew plc (UK) Stryker Corporation (US) Takeda Pharmaceutical Company Limited (Japan) Tego Science (South Korea) Vericel Corporation (US) Zimmer Biomet (US)

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The global regenerative medicine market is projected to reach USD 17.9 billion by 2025 from USD 8.5 billion in 2020, at a CAGR of 15.9% -...

The label expansion includes the addition of longer-term efficacy data demonstrating sustained phenylalanine (Phe) lowering out to three years and over six years of data further supporting a well-characterized safety profile. The labeling was updated to allow individualized maintenance dosage to achieve blood Phe control (blood Phe concentrations less than or equal to 600 mol/L), taking into account patient tolerability to Palynziq and dietary protein intake.

Palynziq is indicated to reduce blood Phe concentrations in adults with phenylketonuria (PKU), who have uncontrolled blood Phe concentrations greater than 600 mol/L on existing management. Palynziq, a PEGylated recombinant phenylalanine ammonia lyase enzyme, is the first and only approved enzyme substitution therapy to target the underlying cause of PKU by helping the body to break down Phe.

Palynziqis BioMarin's second approved treatment for this serious condition. BioMarin also recently announced that the Company had dosed the first participant in the global Phearless Phase 1/2 study of BMN 307, an investigational gene therapy for people with PKU, and potentially its third approved treatment. BMN 307 has Fast Track Designation in the US, as well as Orphan Drug Designation in the U.S. and E.U.

This labeling long term data update is based on efficacy data from an open-label extension study out to three years demonstrating that two-thirds (66%) of the 86 participants treated for at least two years, had a blood Phe level 360 mol/L consistent with the Phe target in American College of Medical Genetics and Genomics (ACMG) recommended guidelines, and 50% of participants had blood Phe levels 120 mol/L at two years.Of the 77 participants treated, 58 (75%), 51 (66%), and 37 (48%) had a blood Phe concentration less than or equal to 600, 360, and 120 mol /L, respectively, at three years of treatment. Additional safety data with over six years of follow up remains consistent with the previous safety profile of Palynziq, irrespective of dose.

PKU is a rare genetic disease that manifests at birth and results in a variety of cumulative toxic effects on the brain. PKU affects approximately 1 in 12,500 live births in the United States each year. PKU is marked by an inability to break down Phe, an amino acid that is found in all forms of protein. Left untreated, high levels of Phe become toxic to the brain and may lead to serious neurological and neuropsychological impairment, affecting a person's ability to think and problem solve, and can lead to depression, anxiety, and behavior disturbance impacting quality of life. Due to the seriousness of these symptoms, infants are screened at birth to ensure that they are diagnosed early and treated to avoid intellectual disability and other complications. Individuals living with PKU require life-long management, including adherence to a challenging and severely restrictive daily diet of medical foods and formula that avoids the ingestion of Phe that is present in most foods.

"BioMarin is pleased that the FDA has recognized the importance of including an additional dosing option to individuals with PKU. Consistent with the label in Europe, Palynziq is now available in the U.S. at doses of up to 60 mg," said Jean-Jacques Bienaim, Chairman and Chief Executive Officer of BioMarin. "BioMarin remains committed to the PKU community and continues to build on our 15 plus years of research and development, which enabled us to deliver the first two PKU drug therapies. We continue our commitment to further understand the safety and efficacy of our treatments for PKU. We are grateful to the study participants, investigators, study staff and BioMarin employees who were essential to enable this label expansion."

"Adding a maximum dose of 60 mg allows more patients to optimize and achieve treatment goals to keep blood Phe levels within the range set in the medical guidelines or within normal range," said Cary Harding, M.D., professor at Oregon Health & Science University and Steering Committee Chair for the Palynziq program. "More than six years of long-term safety data further supports a well-characterized safety profile similar to the initial data and is important information to physicians considering a chronic therapy."

"It is essential to the PKU community to have treatment options that can control Phe levels and provide long term data on safety and efficacy as it becomes available. The additional information added to the Palynziq label, particularly the long-term safety follow-up demonstrates BioMarin's ongoing commitment to people with PKU," said Christine Brown, MS, executive director of the National PKU Alliance. "BioMarin's 15-year focus on innovative medical research to advance the standard of care in PKU with an existing treatment and for new treatments is transforming the way that the community thinks about this rare genetic disease."

BioMarin's 15-Plus Year Commitment to PKU Research

For more than 15 years, BioMarin has been a pioneer in ongoing research to help improve the lives of PKU patients. BioMarin has developed therapies that have been used to treat approximately 7,000 PKU patients around the world. The company has two approved PKU therapies, and an investigational gene therapy BMN 307 is currently in development. BioMarin has conducted 41 clinical studies in PKU and has sponsored 44 external clinical studies. BioMarin researchers have authored 65 publications in medical and scientific journals on PKU and supported another 57 publications by external researchers.

About Phenylketonuria

PKU, or phenylalanine hydroxylase (PAH) deficiency, is a genetic disorder affecting approximately 70,000 diagnosed patients in the regions of the world where BioMarin operates and is caused by a deficiency of the enzyme PAH. This enzyme is required for the metabolism of Phe, an essential amino acid found in most protein-containing foods. If the active enzyme is not present in sufficient quantities, Phe accumulates to abnormally high levels in the blood and becomes toxic to the brain, resulting in a variety of complications including severe intellectual disability, seizures, tremors, behavioral problems and psychiatric symptoms. As a result of newborn screening efforts implemented in the 1960s and early 1970s, virtually all individuals with PKU under the age of 40 in countries with newborn screening programs are diagnosed at birth and treatment is implemented soon after. PKU can be managed with a severe Phe-restricted diet, which is supplemented by low-protein modified foods and Phe-free medical foods; however, it is difficult for most patients to adhere to the life-long strict diet to the extent needed to achieve adequate control of blood Phe levels. Dietary control of Phe in childhood can prevent major developmental neurological toxicities, but poor control of Phe in adolescence and adulthood is associated with a range of neurocognitive disabilities with significant functional impact.

To learn more about PKU and PAH deficiency, please visit http://www.PKU.com. Information on this website is not incorporated by reference into this press release.

About ACMG Guidelines

Practice guidelines issued by ACMG support the need for lifelong management of Phe levels in patients with PKU. The diagnosis and management guidelines were published online in Genetics In Medicine's Advance Online Publication (AOP) service.

The guidelines state that treatment of PKU should be initiated as early as possible and must be continued throughout adulthood and "lifelong," with a goal of maintaining blood levels of Phe for all patients between 120 to 360 micromol/L. Patients treated from the early weeks of life with initial good metabolic control, but who lose that control in later childhood or adult life, may experience both reversible and irreversible neurocognitive and neuropsychiatric consequences.

According to the guidelines "the primary goal of therapy is to lower blood Phe, and any interventions, including medications, or combination of therapies that help to achieve that goal in an individual, without other negative consequences, should be considered appropriate therapy."

Evidence for the guidelines are drawn from two previous independent review processes from the National Institutes of Health (2001) and the Agency for Health Research and Quality (2012). The guidelines can be accessed here.

About Palynziq

Palynziqsubstitutes the deficient phenylalanine hydroxylase (PAH) enzyme in PKU with the PEGylated version of the enzyme phenylalanine ammonia lyase to break down Phe. Palynziqis administered using a dosing regimen designed to facilitate tolerability; Palynziq's safety profile consists primarily of immune-mediated responses, including anaphylaxis, for which robust risk management measures effective in clinical trials are in place.

The dosing and administration of Palynziq follows an induction, titration, and maintenance paradigm. Treatment is individualized to the lowest effective and tolerated dosage. Prescribers may consider increasing from 20 mg to 40 mg in patients who have been on 20 mg once daily continuously for at least 24 weeks and who have not achieved blood Phe control. Prescribers may increase dosage to a maximum of 60 mg once daily in patients who have been on 40 mg once daily continuously for at least 16 weeks and who have not achieved blood Phe control. Prescribers are instructed to discontinue treatment in patients who have not responded after 16 weeks of continuous treatment with the maximum dosage of 60 mg once daily. Periodic blood Phe monitoring is recommended, and patients should be counseled on how to adjust their dietary intake, as needed, based on blood Phe concentrations.

To reach a BioMarin RareConnections case manager, please call, toll-free, 1-866-906-6100 or e-mail[emailprotected]. For more information about Palynziq, please visitwww.palynziq.com. For additional information regarding this product, please contact BioMarin Medical Information at [emailprotected].

INDICATION

PALYNZIQ(pegvaliase-pqpz) is a phenylalanine (Phe)-metabolizing enzyme indicated to reduce blood Phe levels in adult patients with phenylketonuria who have uncontrolled blood Phe levels greater than 600 micromol/L on existing management.

BOXED WARNING: RISK OF ANAPHYLAXIS

WARNINGS AND PRECAUTIONS

Anaphylaxis

Other Hypersensitivity Reactions

ADVERSE REACTIONS

Blood Phenylalanine Monitoring and Diet

DRUG INTERACTIONS

Effect of PALYNZIQ on Other PEGylated Products

USE IN SPECIFIC POPULATIONS

Pregnancy and Lactation

Pediatric Use

Geriatric Use

You are encouraged to report suspected adverse reactions to BioMarin at1-866-906-6100, or to FDA at1-800-FDA-1088orwww.fda.gov/medwatch.

Please see accompanying full Prescribing Information, including Boxed Warning.

About BioMarin

BioMarin is a global biotechnology company that develops and commercializes innovative therapies for people with serious and life-threatening rare disorders. The company's portfolio consists of six commercialized products and multiple clinical and pre-clinical product candidates.

For additional information, please visit http://www.BMRN.com. Information on BioMarin's website is not incorporated by reference into this press release.

Forward-Looking Statements

This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including, without limitation, statements about: expectations regarding the potential impact of Palynziq in the PKU community; the updated Palynziq label allowing individualized maintenance dosage to achieve blood Phe control (blood Phe concentrations less than or equal to 600 micromol/L), taking into account patient tolerability to Palynziq and dietary protein intake; new data supporting Phe lowering and a well-characterized safety profile; the Company's Phase 1/2 Phearless study of BMN 307, including BMN 307 potentially becoming BioMarin's third approved treatment for PKU; and BioMarin's development programs for PKU and BioMarin's ongoing commitment to patients with PKU generally. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others: actions by regulatory agencies, results and timing of current and planned clinical trials of BioMarin's products, the risks related to the commercialization of Palynziq, our ability to manufacture sufficient quantities of Palynziq; and those other risks detailed from time to time under the caption "Risk Factors" and elsewhere in the Company's Securities and Exchange Commission (SEC) filings including the Annual Report on Form10-Q for the quarter ended June 30, 2020, and future filings and reports by the Company. Stockholders are urged not to place undue reliance on forward-looking statements contained in this press release, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation, to update or alter any forward-looking statement, whether as a result of new information, future events, changes in its expectations or otherwise.

BioMarin and Palynziq are registered trademarks of BioMarin Pharmaceutical Inc.

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Debra Charlesworth

BioMarin Pharmaceutical Inc.

BioMarin Pharmaceutical Inc.

(415) 455-7558

(415) 455-7451

SOURCE BioMarin Pharmaceutical Inc.

https://www.biomarin.com/

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BioMarin, Pioneer in Rare Disease Treatments for Phenylketonuria (PKU), Receives FDA Approval of Label Expansion to Allow Maximum Dose of 60 mg for...

New York, Oct. 08, 2020 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Global Soft Tissue Repair Market 2020-2024" - https://www.reportlinker.com/p04796687/?utm_source=GNW Our reports on soft tissue repair market provides a holistic analysis, market size and forecast, trends, growth drivers, and challenges, as well as vendor analysis covering around 25 vendors. The report offers an up-to-date analysis regarding the current global market scenario, latest trends and drivers, and the overall market environment. The market is driven by the rising incidence of accidental injuries, rise in strategic partnerships for distribution of products and reimbursement for soft tissue repair products. In addition, rising incidence of accidental injuries is anticipated to boost the growth of the market as well. The soft tissue repair market analysis includes product segment and geographical landscapes.

The soft tissue repair market is segmented as below: By Product Soft tissue fixation devices and accessories Cell therapy Tissue scaffold

By Geographical Landscapes North America Europe Asia ROW

This study identifies the growing demand for gene therapy as one of the prime reasons driving the soft tissue repair market growth during the next few years. Also, increasing strategic alliances and the development of novel products will lead to sizable demand in the market.

The analyst presents a detailed picture of the market by the way of study, synthesis, and summation of data from multiple sources by an analysis of key parameters. Our soft tissue repair market covers the following areas: Soft tissue repair market sizing Soft tissue repair market forecast Soft tissue repair market industry analysis

Read the full report: https://www.reportlinker.com/p04796687/?utm_source=GNW

About ReportlinkerReportLinker is an award-winning market research solution. Reportlinker finds and organizes the latest industry data so you get all the market research you need - instantly, in one place.

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The Global Soft Tissue Repair Market is expected to grow by USD 10.44 bn during 2020-2024, progressing at a CAGR of 11% during the forecast period -...

WORCESTER, Mass., Oct. 08, 2020 (GLOBE NEWSWIRE) -- Mustang Bio, Inc. (Mustang) (NASDAQ: MBIO), a clinical-stage biopharmaceutical company focused on translating todays medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases, today announced that initial Phase 1 data on MB-105, a PSCA-targeted CAR T administered systemically to patients with PSCA-positive metastatic castration-resistant prostate cancer (mCRPC), will be presented at the virtual 27th Annual Prostate Cancer Foundation Scientific Retreat, which is being held October 20 23, 2020.

Manuel Litchman, M.D., President and Chief Executive Officer of Mustang, said, We are pleased that City of Hope will be presenting initial data from the Phase 1 trial of MB-105 at the virtual 27th Annual Prostate Cancer Foundation Scientific Retreat. MB-105 is our CAR T cell therapy that is being studied for the potential treatment of prostate cancer. We look forward to continuing to progress this PSCA-targeted CAR T program.

Details of the presentation are as follows:

Title: Clinical Development of PSCA-targeted CAR T cell therapy for mCRPCSession Date and Time:Friday, October 23, 2020, 11:50 a.m. PTPresenter: Tanya Dorff, M.D., Associate Professor, Department of Medical Oncology & Experimental Therapeutics,Head, Genitourinary Cancer Program, City of Hope, Duarte, CA

For more information, please visit the 27th Annual Prostate Cancer Foundation Scientific Retreat website at https://www.pcf.org/scientific-retreat/27th-annual/.

About MB-105 (PSCA CAR T technology)MB-105 was developed in the laboratory of Saul Priceman, Ph.D., assistant professor in City of Hopes Department of Hematology & Hematopoietic Cell Transplantation and a scientist in the T Cell Therapeutics Research Laboratory led by Stephen Forman, M.D., leader of City of Hopes Hematologic Malignancies and Stem Cell Transplantation Institute and the laboratorys director.

The Phase 1 clinical trial of MB-105, one of the first chimeric antigen receptor T cell (CAR T) trials for prostate cancer in the nation, can enroll up to 33 patients. Its primary endpoints are to define the side effects and the best dose of the prostate stem cell antigen (PSCA) CAR T cells in treating patients with PSCA-positive metastatic castration-resistant prostate cancer (mCRPC). Secondary endpoints include assessing the expansion and persistence of PSCA CAR T cells, the clinical response based on Prostate Cancer Working Group 3 (PCWG3) criteria, the survival outcomes and serum cytokine profiles in peripheral blood pre- and post-therapy, as well as describing the PSCA expression level on tumor cells prior to CAR T cell infusion and the relationship it may have with disease response and toxicities. For more information on this Phase 1 trial, please visit http://www.clinicaltrials.gov using identifier NCT03873805.

About Mustang BioMustang Bio, Inc. is a clinical-stage biopharmaceutical company focused on translating todays medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases. Mustang aims to acquire rights to these technologies by licensing or otherwise acquiring an ownership interest, to fund research and development, and to outlicense or bring the technologies to market. Mustang has partnered with top medical institutions to advance the development of CAR T therapies across multiple cancers, as well as a lentiviral gene therapy for X-linked severe combined immunodeficiency (XSCID), also known as bubble boy disease. Mustang is registered under the Securities Exchange Act of 1934, as amended, and files periodic reports with the U.S. Securities and Exchange Commission (SEC). Mustang was founded by Fortress Biotech, Inc. (NASDAQ: FBIO). For more information, visit http://www.mustangbio.com.

ForwardLooking StatementsThis press release may contain forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, each as amended. Such statements include, but are not limited to, any statements relating to our growth strategy and product development programs and any other statements that are not historical facts. Forward-looking statements are based on managements current expectations and are subject to risks and uncertainties that could negatively affect our business, operating results, financial condition and stock value. Factors that could cause actual results to differ materially from those currently anticipated include: risks relating to our growth strategy; our ability to obtain, perform under, and maintain financing and strategic agreements and relationships; risks relating to the results of research and development activities; risks relating to the timing of starting and completing clinical trials; uncertainties relating to preclinical and clinical testing; our dependence on third-party suppliers; our ability to attract, integrate and retain key personnel; the early stage of products under development; our need for substantial additional funds; government regulation; patent and intellectual property matters; competition; as well as other risks described in our SEC filings. We expressly disclaim any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in our expectations or any changes in events, conditions or circumstances on which any such statement is based, except as required by law, and we claim the protection of the safe harbor for forward-looking statements contained in the Private Securities Litigation Reform Act of 1995.

Company Contacts:Jaclyn Jaffe and William BegienMustang Bio, Inc.(781) 652-4500ir@mustangbio.com

Investor Relations Contact:Daniel FerryLifeSci Advisors, LLC(617) 430-7576daniel@lifesciadvisors.com

Media Relations Contact:Tony Plohoros6 Degrees(908) 591-2839tplohoros@6degreespr.com

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Mustang Bio Announces MB-105 Data Selected for Presentation at the Virtual 27th Annual Prostate Cancer Foundation Scientific Retreat - GlobeNewswire

- Kuick Research Report Analyzes Clinical & Commercial Opportunity In Global Bispecific Antibodies Market

NEW DELHI, Oct. 7, 2020 /PRNewswire/ --

"Global Bispecific Antibody Market Opportunity, Drug Sales & Clinical Trials Insight 2026" Report Highlights:

Download Report:

https://www.kuickresearch.com/report-global-usa-europe-japan-bispecific-antibody-antibodies-therapeutics-therapy-market-sales-size--trends-clinical-trials-emicizumab-hemlibra-blincyto--blinatumomab-sales

Despite having several hundred clinical success stories of different immunotherapies against solid and advanced cancers, the overall response rate of the patients has reached approximately 50% even when the patients fit completely under all the criteria of treatment. To pave with the clinical failures, researchers have developed and evaluated bispecific therapeutic antibodies, that are combination of two binding specificities leading to improve selectivity as well as efficacy of antibody-based treatment for cancer. The arrival of bispecific antibody for cancer treatment has estimated to be of great potential for wide range of applications in cancer treatment. The current biotherapeutic market for cancer treatment and prevention is estimated to be dominated by antibody drugs, where bispecific antibody represents a novel ally for the next-generation of cancer therapy.

In the past few years, the remarkable progress that has been made with respect to understanding the important hallmarks of the cancer progression and main epitopes involved in it have led to paradigmatic development in the bispecific antibody capable of offering novel promises to the patients who haven't respond to any medical therapy for cancer. The practice of bispecific antibody is not new i.e. this version of cancer therapeutics is a modified version of monoclonal antibody. By observing the increasing incidence of cancer, the management associated with the bispecific antibody has started to develop several strategies important for clinical management of the cancer in the present and in the future.

As per the analysis conducted, it is estimated that the bispecific antibody therapeutics market is growing at a double-digit rate, thus boosting the overall cancer therapeutics market to extreme, especially when compared with other immunotherapies. Further, trends and opportunities coupled within the market i.e. rise in the number of cancer cases, government favorable policies and many others are tending to drive the growth of the market. In our viewpoint, novel alliances developed by major key players of the market and local manufacturers are estimated to be playing important role for delivering tremendous opportunities for the researchers, patients, stakeholders and the payers.

The analysis of the Global bispecific antibody drug market represents that the market is completely focused on providing a better structure to the overall therapeutics market and offer the pharmaceutical industry a broad perspective of an efficient therapy for every cancer type. The research report provides analysis about the assess growth potential of the market by verifying various driving forces, in combination withall the important trends, opportunities and challenges. In addition to it, the research report also provides a long-term outlook for the industry and various other important measures that have helped the market to attract thousands of investors and local drug manufacturers. Lastly, the report also profiles various pharmaceutical companies of the dominating markets that have been boosting the R&D sector of the market.

Table of Contents

1. Introduction to Bispecific Monoclonal Antibody

1.1 Overview of Monoclonal Antibody

1.2 Overview of Bispecific Monoclonal Antibody

1.3 Advantage of Bispecific Antibodies upon Monospecific Monoclonal Antibodies

2. The Engineering & Development of Bispecific Antibodies

2.1 Bispecific Antibodies Similar to IgG

2.1.1 Quadroma (Hybrid Hydromas) Approach

2.1.2 "Knobs Into Holes" Approach

2.1.3 CrossMab Approach

2.1.4 Dual-Variable-Domain Immunoglobulin Approach

2.2 Small Bispecific Antibodies

2.2.1 Bispecific Diabodies

2.2.2 Bispecific T-Cell Engager Antibodies (BiTEs)

3. Mechanism Of Action Of Bispecific Antibody

3.1 Trifunctional Antibody: Catumaxomab (Removab)

3.2 Blinatumomab

4. Applications of Bispecific Antibodies in Diagnostic & Treatment

4.1 BsMAb for Diagnosis of Bacterial & Viral Infectious Diseases

4.2 BsMAb for Cancer Diagnostic

4.3 BsAbs Blocking Signaling Pathways

4.4 BsAbs Targeting Tumor Angiogenesis

4.5 Specific Delivery of Effector Compounds to Targets

4.6 Bispecific Antibodies & Gene Therapy

5. Global Bispecific Antibody Market Outlook

5.1 Current Market Scenario

5.2 Availability of Bispecific Antibodies

5.2.1 Catumaxomab (Removab)

5.2.2 Blinatumomab

5.2.3 Emicizumab

5.2.4 Duligotumab

5.2.5 SAR 156597

6. Approved Bispecific Antibody Dosage & Price Analysis

6.1 Blincyto

6.2 Hemlibra

7. Approved Bispecific Antibody Sales Analysis

7.1 Blincyto

7.2 Hemlibra

8. Global Bispecific Antibodies Clinical Pipeline Overview

8.1 By Phase

8.2 By Country/Region

8.3 By Company

8.4 By Indication

8.5 Orphan Designated Bispecific Antibodies

9. Global Bispecific Antibodies Clinical Pipeline By Company, Indication & Phase

9.1 Research

9.2 Preclinical

9.3 Phase-I

9.4 Phase-I/II

9.5 Phase-II

9.6 Phase-II/III

9.7 Phase-III

10. Marketed Bispecific Antibodies Clinical Insight

10.1 Blinatumomab - Amgen

10.2 Emicizumab - Chugai Pharmaceutical

11. Global Bispecific Antibody Market Dynamics

11.1 Favorable Market Parameters

11.2 Commercialization Challenges

12. Global Bispecific Antibody Market Future Prospects

13. Competitive Landscape

13.1 Ablynx

13.2 Adimab

13.3 Affimed Therapeutics

13.4 Amgen

13.5 AstraZeneca (MedImmune)

13.6 Chugai Pharmaceutical

13.7 Eli Lilly

13.8 EMD Serono

13.9 Emergent BioSolutions

13.10 Genentech

13.11 Genmab

13.12 Immunomedics

13.13 Jounce Therapeutics

13.14 MacroGenics

13.15 Merus

13.16 Neovii Biotech

13.17 NovImmune SA

13.18 OncoMed Pharmaceuticals

13.19 Pieris

13.20 Regeneron Pharmaceuticals

13.21 Roche

13.22 Sanofi

Contact:Neeraj Chawla[emailprotected]+91-9810410366

Logo: https://mma.prnewswire.com/media/1245952/Kuick_Research_Logo.jpg

SOURCE Kuick Research

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Global Bispecific Antibodies Therapy Blincyto Hemlibra Therapeutics Market Size To Witness 118% Growth To Reach US$ 10 Billion By 2026 - PR Newswire...

Newswise PHILADELPHIA Picture a future where embedded medical devices not only treat and teach patients, but learn from them. Imagine chronic pain being managed without the negative side effects of opioids. Penn Medicine researchers are working to make those scenarios a reality. These initiatives are among innovative new National Institutes of Health (NIH) funded efforts that are mapping the future of medicine.

The NIH has selected two researchers from the Perelman School of Medicine at the University of Pennsylvania to receive its prestigious Directors Awards, part of the NIH Common Funds High-Risk, High-Reward Research Program honoring exceptionally creative scientists. Brian Litt, MD, a professor of Neurology, Neurosurgery, and Bioengineering, was honored with a Pioneer Award for $5.6 million, supporting novel neurodevice research. Gregory Corder, PhD, an assistant professor of Psychiatry and Neuroscience, was selected as a New Innovator Award winner, receiving $2.4 million for research investigating the mechanisms of chronic pain.

This NIH initiative, designed to fuel research endeavors that are more opened-ended and have a potentially broader effect on scientific understanding compared to more traditional research, awards these to scientists to support research over a five-year period. The 2020 Penn recipients are among 85 awardees nationally:

Pioneer Award Ghost in the Machine: Melding Brain, Computer and Behavior

The Pioneer Award challenges investigators to pursue new research directions and develop groundbreaking, high-impact approaches to a broad area of biomedical or behavioral science. This award supports Litts work to develop a new generation of autonomous neurodevicesimplanted machines that can question, record, and combine learning algorithms based on neurological signals and feedback to act and alter human behavior on the fly.

In epilepsy, for example, these devices would predict and prevent seizures; in Parkinsons patients, implants will measure and communicate with patients to improve mobility, reduce tremor and enhance responsiveness. Other implants might improve hearing or psychiatric symptoms by querying patient perceptions, feelings, and altering stimulation patterns algorithmically to improve them. The loop is closed in real time, so the host can change their behavior based upon device feedback to improve their health.

Imagine this: A 30 year-old veteran walks into a bar. None of the patrons are aware of the anti-seizure device in his brain, or the traumatic brain injury that requires it. After he drinks a beer, his phone vibrates with a text from the implantable device asking what hes doing and sharing his probability of a seizure has increased. And with a quick explanation from the veteran, the device stimulates and suggests avoiding a second beverage, Litt explains. Were working towards this future, with the help of the Pioneer Award.

This is a paradigm shift from todays simple devices, which rely on physicians to give device feedback to patients and change simple parameters by hand during occasional office visits. Litts goal is to build a foundation for responsive implants that can collaborate with hosts, linking human experience and perception to machine algorithms, actions, and therapy, predicting and preventing events before they start. Not only will the patient teach the device, but the device will teach the patient.

Grant ID: DP1 NS122038-01.

New Innovator Award Harnessing Cortical Neuromodulation to Disrupt Pain Perception

The New Innovator Award supports unusually innovative research from early career investigators who have not yet received a research project grant or equivalent NIH grant. The award will support Corders efforts to research the mechanisms of chronic paina major health crisis in the United States, affecting millions, and a driver of the opioid epidemic.

Corders goal is to identify which parts of the brain are important for pain perception and which circuits impact pain relief from opioids. He hopes to decode how this neural activity evolves during chronic pain. Once the brain circuits and pathways that contribute to the suffering and perception of pain are identified, they can be targeted for potential therapeutics which could be more effective at reducing pain and without the addictive elements of prescription opioids.

Corder envisions these next generation pain therapeutics leveraging viral-delivered cell-specific gene therapies to disrupt the pain-processing circuits in the cortex. This would will allow patients to sense pain but without the unpleasant aversion.

We currently have a limited understanding of the neural pathways in the brain that contribute to pain, which has been a significant barrier for treating pain efficiently, without negative side effects. But, if we can identify and understand these circuits, we can then try to rewrite the neural code of pain, Corder said. Picture being able to specifically target the one desired brain region or circuit that processes pain in order to provide pain relief. This research will help us lay the groundwork for new classes of therapeutics, which could have a profound and broad impact for treating patients with chronic pain, while reducing the burden of the national opioid crisis.

Grant ID: DP2 GM140923-01.

###

Penn Medicineis one of the worlds leading academic medical centers, dedicated to the related missions of medical education, biomedical research, and excellence in patient care. Penn Medicine consists of theRaymond and Ruth Perelman School of Medicine at the University of Pennsylvania (founded in 1765 as the nations first medical school) and theUniversity of Pennsylvania Health System, which together form a $8.6 billion enterprise.

The Perelman School of Medicine has been ranked among the top medical schools in the United States for more than 20 years, according toU.S. News & World Report's survey of research-oriented medical schools. The School is consistently among the nation's top recipients of funding from the National Institutes of Health, with $494 million awarded in the 2019 fiscal year.

The University of Pennsylvania Health Systems patient care facilities include: the Hospital of the University of Pennsylvania and Penn Presbyterian Medical Centerwhich are recognized as one of the nations top Honor Roll hospitals byU.S. News & World ReportChester County Hospital; Lancaster General Health; Penn Medicine Princeton Health; and Pennsylvania Hospital, the nations first hospital, founded in 1751. Additional facilities and enterprises include Good Shepherd Penn Partners, Penn Medicine at Home, Lancaster Behavioral Health Hospital, and Princeton House Behavioral Health, among others.

Penn Medicine is powered by a talented and dedicated workforce of more than 43,900 people. The organization also has alliances with top community health systems across both Southeastern Pennsylvania and Southern New Jersey, creating more options for patients no matter where they live.

Penn Medicine is committed to improving lives and health through a variety of community-based programs and activities. In fiscal year 2019, Penn Medicine provided more than $583 million to benefit our community.

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Penn Medicine Researchers Receive Prestigious National Institutes of Health Directors Awards - Newswise

LONDON, Oct. 06, 2020 (GLOBE NEWSWIRE) -- (Oncology Drugs Companies Included: F. Hoffmann-La Roche AG, Novartis AG, Bristol-Myers Squibb Company, Merck & Co., Inc., Johnson & Johnson)

The global oncology/cancer drugs market size reached a value of nearly $167.9 billion in 2019, having increased at a compound annual growth rate (CAGR) of 9.8% since 2015. The oncology market size 2020 is expected to decline from $167.9 billion in 2019 to $149.9 billion in 2020 at a rate of -11%. The decline is mainly due to lockdown and social distancing norms imposed by various countries and economic slowdown across countries owing to the COVID-19 outbreak and the measures to contain it. The market is then expected to grow slightly from $201.1 billion in 2021 to $284.5 billion in 2023 at a CAGR of 9.7%. North America has the largest cancer drug market share, accounting for 41.2% of the total market.

The Business Research Companys report titled Oncology Drugs Market - Opportunities And Strategies - Global Forecast To 2030 covers major oncology drugs companies, oncology drugs market share by company, oncology drugs manufacturers, oncology drugs infrastructure market size, and oncology drugs market forecasts. The report also covers the global oncology drugs market and its segments. The cancer market by type is segmented into drugs for lung cancer, pancreatic cancer, breast cancer, prostate cancer, ovarian cancer, colorectal cancer, gastric cancer, kidney cancer, brain tumor, thyroid cancer, skin cancer, bladder cancer, cervical cancer, blood cancer and others, by drug class type into targeted therapy, immunotherapy (biologic therapy), chemotherapy and hormonal therapy, by distribution channel into hospital pharmacies, retail pharmacies/drug stores and others, by route of administration into oral, parental and others, by drug classification into branded drugs and generic drugs.

Request For A Sample Of The Global Oncology Drugs Market Report:

https://www.thebusinessresearchcompany.com/sample.aspx?id=3440&type=smp

Companies in the oncology drugs market are increasing their product innovation through strategic collaborations. To sustain in the increasingly competitive market, organizations are developing innovative products as well as sharing skills and expertise with other such enterprises. While oncology drug companies have long collaborated with each other as well as with academic and research institutions in this market by way of partnerships, in or out licensing deals, this trend has been increasing over the recent years.

New technologies are being implemented in the cancer drug market, such as artificial intelligence in the research and development process, as well as 3D printing devices to mimic the human body for trials and testing of the drugs developed. Companies are also investing in technologies to develop next generation biologics such as antibody drug conjugates, bispecifics, fusion proteins, cell and gene therapy that will be more effective as they are expected to have better potency against the target disease as well as have the ability to treat more than one aspect of the disease. Over the last few years, there has been a significant rise in accelerated approval of cell and gene therapies for cancer treatment by regulatory bodies across the globe. CRISPR technology can be used to discover the non-coding cancer genome. CRISPR-Cas9 genome editing reduces the processing associated with the generation of cell line and animal models of cancer and complex generations. Thus, it generates a better cancer model for target validation and drug evaluation. CRISPRCas9 is accelerating the different stages of oncology drug discovery including target identification, validation and deconvolution, drug synthesis, assessment of drug sensitivity and resistance.

With innovation through technology and rising focus on development of new drugs, the oncology drugs market trends ought to bring advances in cancer therapies in the forecasted period.

Oncology Drugs Market - Opportunities And Strategies - Global Forecast To 2030 is one of a series of new reports from The Business Research Company that provide oncology drugs market overviews, oncology drugs market analyze and forecast oncology drugs market size and growth for the whole market, oncology drugs market segments and oncology drugs market geographies, oncology drugs market trends, oncology drugs market drivers, oncology drugs market restraints, oncology drugs market leading competitors revenues, profiles and market shares in over 1,000 industry reports, covering over 2,500 market segments and 60 geographies.

The report also gives in-depth analysis of the impact of COVID-19 on the market. The reports draw on 150,000 datasets, extensive secondary research, and exclusive insights from interviews with industry leaders. A highly experienced and expert team of analysts and modelers provides market analysis and forecasts. The reports identify top countries and segments for opportunities and strategies based on market trends and leading competitors approaches.

Here Is A List Of Similar Reports By The Business Research Company:

Clinical Oncology Next Generation Sequencing Global Market Report 2020-30: COVID 19 Growth and Change

Interventional Oncology Devices Global Market Report 2020-30: COVID 19 Growth and Change

Cancer Diagnostics Global Market Report 2020-30: COVID 19 Growth And Change

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Oncology Market Size 2020 Particularly Prone To Disruption During Pandemic In The Global Oncology Market - GlobeNewswire

DELHI, India, Oct. 07, 2020 (GLOBE NEWSWIRE) -- "Global Bispecific Antibody Market Opportunity, Drug Sales & Clinical Trials Insight 2026" Report Highlights:

Download Report:

https://www.kuickresearch.com/report-global-usa-europe-japan-bispecific-antibody-antibodies-therapeutics-therapy-market-sales-size--trends-clinical-trials-emicizumab-hemlibra-blincyto--blinatumomab-sales

Despite having several hundred clinical success stories of different immunotherapies against solid and advanced cancers, the overall response rate of the patients has reached approximately 50% even when the patients who fit completely under all the criteria of treatment. To pave with the clinical failures, researchers have developed and evaluated bispecific therapeutic antibodies, that are combination of two binding specificities leading to improve selectivity as well as efficacy of antibody-based treatment for cancer. The arrival of bispecific antibody for cancer treatment has estimated to be of great potential for wide range of applications in cancer treatment. The current biotherapeutic market for cancer treatment and prevention is estimated to be dominated by antibody drugs, where bispecific antibody represents a novel ally for the next-generation of cancer therapy.

In the past few years, the remarkable progress that has been made with respect to understanding the important hallmarks of the cancer progression and main epitopes involved in it have led to paradigmatic development in the bispecific antibody capable of offering novel promises to the patients who havent respond to any medical therapy for cancer. The practice of bispecific antibody is not new i.e. this version of cancer therapeutics is a modified version of monoclonal antibody. By observing the increasing incidence of cancer, the management associated with the bispecific antibody has started to develop several strategies important for clinical management of the cancer in the present and in the future.

As per the analysis conducted, it is estimated that the bispecific antibody therapeutics market is growing at a double-digit rate, thus boosting the overall cancer therapeutics market to extreme, especially when compared with other immunotherapies. Further, trends and opportunities coupled within the market i.e. rise in the number of cancer cases, government favorable policies and many others are tending to drive the growth of the market. In our viewpoint, novel alliances developed by major key players of the market and local manufacturers are estimated to be playing important role for delivering tremendous opportunities for the researchers, patients, stakeholders and the payers.

The analysis of the Global bispecific antibody drug market represents that the market is completely focused on providing a better structure to the overall therapeutics market and offer the pharmaceutical industry a broad perspective of an efficient therapy for every cancer type. The research report provides analysis about the assess growth potential of the market by verifying various driving forces, in combination with the all the important trends, opportunities and challenges. In addition to it, the research report also provides a long-term outlook for the industry and various other important measures that have helped the market to attract thousands of investors and local drug manufacturers. Lastly, the report also profiles various pharmaceutical companies of the dominating markets that have been boosting the R&D sector of the market.

Neeraj Chawla neeraj@kuickresearch.com +91-9810410366

Table of Contents

1. Introduction to Bispecific Monoclonal Antibody

1.1 Overview of Monoclonal Antibody 1.2 Overview of Bispecific Monoclonal Antibody 1.3 Advantage of Bispecific Antibodies upon Monospecific Monoclonal Antibodies

2. The Engineering & Development of Bispecific Antibodies

2.1 Bispecific Antibodies Similar to IgG

2.1.1 Quadroma (Hybrid Hydromas) Approach 2.1.2 Knobs Into Holes Approach 2.1.3 CrossMab Approach 2.1.4 Dual-Variable-Domain Immunoglobulin Approach

2.2 Small Bispecific Antibodies

2.2.1 Bispecific Diabodies 2.2.2 Bispecific T-Cell Engager Antibodies (BiTEs)

3. Mechanism Of Action Of Bispecific Antibody

3.1 Trifunctional Antibody: Catumaxomab (Removab) 3.2 Blinatumomab

4. Applications of Bispecific Antibodies in Diagnostic & Treatment

4.1 BsMAb for Diagnosis of Bacterial & Viral Infectious Diseases 4.2 BsMAb for Cancer Diagnostic 4.3 BsAbs Blocking Signaling Pathways 4.4 BsAbs Targeting Tumor Angiogenesis 4.5 Specific Delivery of Effector Compounds to Targets 4.6 Bispecific Antibodies & Gene Therapy

5. Global Bispecific Antibody Market Outlook

5.1 Current Market Scenario

5.2 Availability of Bispecific Antibodies 5.2.1 Catumaxomab (Removab) 5.2.2 Blinatumomab 5.2.3 Emicizumab 5.2.4 Duligotumab 5.2.5 SAR 156597

6. Approved Bispecific Antibody Dosage & Price Analysis

6.1 Blincyto 6.2 Hemlibra

7. Approved Bispecific Antibody Sales Analysis

7.1 Blincyto 7.2 Hemlibra

8. Global Bispecific Antibodies Clinical Pipeline Overview

8.1 By Phase 8.2 By Country/Region 8.3 By Company 8.4 By Indication 8.5 Orphan Designated Bispecific Antibodies

9. Global Bispecific Antibodies Clinical Pipeline By Company, Indication & Phase

9.1 Research 9.2 Preclinical 9.3 Phase-I 9.4 Phase-I/II 9.5 Phase-II 9.6 Phase-II/III 9.7 Phase-III

10. Marketed Bispecific Antibodies Clinical Insight

10.1 Blinatumomab - Amgen 10.2 Emicizumab - Chugai Pharmaceutical

11. Global Bispecific Antibody Market Dynamics

11.1 Favorable Market Parameters 11.2 Commercialization Challenges

12. Global Bispecific Antibody Market Future Prospects

13. Competitive Landscape

13.1 Ablynx 13.2 Adimab 13.3 Affimed Therapeutics 13.4 Amgen 13.5 AstraZeneca (MedImmune) 13.6 Chugai Pharmaceutical 13.7 Eli Lilly 13.8 EMD Serono 13.9 Emergent BioSolutions 13.10 Genentech 13.11 Genmab 13.12 Immunomedics 13.13 Jounce Therapeutics 13.14 MacroGenics 13.15 Merus 13.16 Neovii Biotech 13.17 NovImmune SA 13.18 OncoMed Pharmaceuticals 13.19 Pieris 13.20 Regeneron Pharmaceuticals 13.21 Roche 13.22 Sanofi

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Global Bispecific Antibodies Therapy Blincyto Hemlibra Antibody Therapeutics Market Size To Witness 118% Growth To Reach US$ 10 Billion By 2026 -...

The FDA has lifted the clinical hold on a phase 1/2 clinical trial of Solid Biosciences gene therapy treatment for Duchenne muscular dystrophy (DMD). Solid Bio secured clearance to resume dosing in the trial after making manufacturing changes to cut the number of viral particles given to patients.

SGT-001, the adeno-associated viral (AAV) vector-mediated gene transfer therapy being tested in the phase 1/2 trial, has suffered a series of setbacks since entering the clinic, most recently when the FDA put the study on hold in response to a case of acute kidney injury. The FDA imposed the hold 11 months ago. In July, Solid Bio said the FDA wanted to see more data before lifting the hold.

The request led Solid Bio to share further information on its gene therapy manufacturing process and its latest safety and efficacy data. The additional information proved sufficient to persuade the FDA to lift the clinical hold.

From Concept to Market: Overcoming the Challenges of Manufacturing and Clinical Trials

Learn how CRO/CDMOs successfully address operational and regulatory challenges for pharmaceutical and biotechnology clients; and how this can make the difference between study success or failure.

Solid Bio will resume dosing using a gene therapy made under a revised manufacturing process. The new process is intended to remove most empty viral capsids, thereby enabling Solid Bio to cut total viral load without reducing the dose. The focus on viral load reflects concerns systemic delivery of AAV vectors can damage organs and cause inflammation.

To get the FDA to lift the hold, Solid Bio shared data from a quantitative, in vitro microdystrophin expression assay designed to show the comparability of SGT-001 manufactured under the old and new processes. Solid Bio shared those results in response to the FDAs request for information in July.

The biotech is taking other precautions to manage the potential risk posed by SGT-001. Solid Bio has capped the maximum weight of the first two patients to receive SGT-001 after the hold lifts at 18 kg. As the dose of SGT-001 is determined by weight, heavier patients receive more vector genomes. The adverse events seen in some gene therapy trials, such as the deaths in Audentes Therapeutics trial, have happened in patients who were heavier and therefore received a higher viral load.

Solid Bio is further mitigating the potential for SGT-001 to cause harm by amending the protocol to include the prophylactic use of eculizumab, the anti-complement inhibitor sold by Alexion as Soliris, and C1 esterase inhibitor, while also increasing the prednisone dose in the month after treatment.

The protocol changes position Solid Bio to resume its pursuit of DMD gene therapy leader Sarepta Therapeutics, which suffered a setback of its own last month when the FDA asked it to use an extra potency assay in a planned clinical trial. Pfizer is also in the race but, like Solid Bio, has run into safety issues that could give Sarepta an edge.

Shares in Solid Bio, which had slumped to $2 apiece, rose 70% in response to the end of the hold.

The rest is here:
FDA lifts clinical hold on Solid Bio gene therapy trial - FierceBiotech

With ongoing advances in science and technology, the cell and gene therapy pipeline has grown especially robust over the past few years. At present, ClinicalTrials.gov shows more than 4,500 active gene therapy trials globally. In the United States, McKinsey experts expect to see 10 to 20 cell and gene therapy approvals per year over the next five years.

This rise in supply has created a heightened demand for contract development and manufacturing organizations (CDMOs) with biotech expertise. CDMOs typically supply materials and handle production and manufacturing, allowing life sciences companies to focus on innovation and marketing.

The bottleneck stems from a shortage of CDMOs with gene therapy expertise and resources. Considering the critical need for safe, effective gene therapies and the rapid pace of development, it's important for pharma and biopharma to find a CDMO with both gene therapy capabilities and availability to take on new partners nownot 18 months from now.

"Full-service CDMOs that can assist with both development and manufacturing are in highest demand,"said Richard Welch, PhD, vice president, development services for Emergent BioSolutions, a global CDMO and specialty life sciences company headquartered in Gaithersburg, Maryland. "As pharma and biopharma companies move from early phase to late phase, CDMOs need experience with process characterization and process validation as well as commercial production and supply chain."

"The supply chain is much more complex,"added Tarek Abdel-Gawad, senior director of commercial strategy for Emergent BioSolutions. "You aren't just growing cells. You're ensuring viruses, helper viruses, and plasmid DNA work together to produce the molecule of choice. Few companies have the capabilities, equipment, and GMP expertise."

Much of the gene therapy development as of late has stemmed from smaller biotech companies or research universities according to a McKinsey report. Large pharmaceutical companies may partner with these organizations on rare disease or oncology treatments two therapeutic areas where much of the research lies.

Many small to midsize companies have the idea and investor support, but do not have the employees, infrastructure or manufacturing space. "A CDMO is a good partner in those cases,"said Mukesh Mayani, PhD, principal scientist, gene therapy at Sanofi. "You can test your hypotheses and work with a CDMO that has the platform, the people, and the preclinical models. This arrangement speeds up the timeline and allows these innovative companies to focus on other modalities and molecules."

Pharma and biopharma companies of all sizes can learn from this "single-source"approach. Partnering with a CDMO earlier in the processfrom preclinical development through packagingfrees up resources to focus on innovation and communication.

"It is neither simple nor cheap to develop and manufacture gene therapies,"said Dr. Welch. "A CDMO has the built-in skill set to grow viruses at the densities necessary to meet early-phase studies while hitting safety margins. With the clinical trial failure rate as high as it is, working with a CDMO that has experience in different technologies and products makes for a more efficient, cost-effective process."

Although there is a high demand now for CDMOs with gene therapy expertise, the market is quickly growing. According to Grand View Research, the CDMO market is expected to grow from $115.6 billion in 2020 to $157.7 billion in 2025, outpacing the pharmaceutical industry as a whole. New cell and gene therapy CDMOs are emerging and established CDMOs are expanding capabilities.

Before you start your CDMO search, consider the following two factors:

When vetting CDMOs for your gene therapy studies, consider the strengths and weaknesses of your company as well as your potential CDMO partner. A few points to consider include:

As gene therapy research continues to expand, innovators in this space will need CDMOs with highly specific expertise, facilities, and equipment. Choose a partner that can assist from the earliest phases of product development all the way to commercialization.

Capra, Emily, et al. "Gene therapy coming of age: Opportunities and challenges to getting ahead."McKinsey, October 2, 2019

Link:
Gene therapy solution: The value of a CDMO as your end-to-end partner - BioPharma Dive

SAN RAFAEL, Calif., Oct. 2, 2020 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN), a pioneer in developing treatments for phenylketonuria (PKU) and gene therapies, announced today that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation toBMN 307, an investigational gene therapy for the treatment of individuals with PKU.

Fast Track designation is designed to facilitate the development and expedite the review of drugs to treat serious conditions and fulfill an unmet medical need, enabling drugs to reach patients earlier. Clinical programs with Fast Track designation may benefit from early and frequent communication with the FDA throughout the regulatory review process. These clinical programs may also be eligible to apply for Accelerated Approval and Priority Review if relevant criteria are met, as well as Rolling Review, which means that completed sections of the Biologic License Application can be submitted for review before the entire FDA application is complete. Both the FDA and European Medicines Agency have granted BMN 307 Orphan Drug Designation.

"Fast Track designation combined with our ability to conduct our clinical studies incorporating material manufactured using a commercial-ready process will further facilitate rapid clinical development of BMN 307 gene therapy," said Hank Fuchs, M.D., President, Worldwide Research and Development at BioMarin. "We are looking forward to working closely with the FDA, as well as other health agencies, to evaluate the safety and efficacy of this promising investigational gene therapy as we continue our unwavering 15-year commitment to advance the standard of care for people with PKU."

PKU is a rare genetic disease that manifests at birth and is marked by an inability to break down Phe, an amino acid that is commonly found in many foods. Left untreated, high levels of Phe become toxic to the brain and may lead to serious neurological and neuropsychological issues, affecting a person's ability to think and problem solve, and can lead to depression, anxiety, and behavior disturbance impacting quality of life. Due to the seriousness of these symptoms, in many countries, infants are screened at birth to ensure early diagnosis and treatment to avoid intellectual disability and other complications. According to treatment guidelines, PKU patients should maintain lifelong control of their Phe levels.

BMN 307 Clinical Program

Last week, BioMarin announced that it had dosed the first participant in the global Phearless Phase 1/2 study with BMN 307, an AAV5-phenylalanine hydroxylase (PAH) gene therapy designed to normalize blood phenylalanine (Phe) concentration levels in patients with PKU by inserting a correct copy of the PAH gene into liver cells. BMN 307 will be evaluated to determine safety and whether a single dose of treatment can restore natural Phe metabolism, increase plasma Phe levels, and enable a normalization of diet in patients with PKU.BioMarin is conducting this study with material manufactured with a commercial-ready process to facilitate rapid clinical development and potentially support approval. BMN 307 represents a potential third PKU treatment option in BioMarin's PKU franchise and a second gene therapy development program.

BioMarin's clinical program is composed of two key studies. Phearless, a Phase 1/2 study, will evaluate the safety, efficacy, and tolerability of a single intravenous administration of BMN 307 in patients with PKU. The study consists of a dose-escalation phase, followed by a cohort expansion phase once an initially efficacious dose has been demonstrated. In addition, BioMarin is sponsoring an observational study, Phenom, which includes patients with PKU to measure both established and new markers of disease and clinical outcomes over time.

BioMarin's 15-Plus Year Commitment to PKU Research

For more than 15 years, BioMarin has been a pioneer in ongoing research to help improve the lives of PKU patients. BioMarin has developed therapies that have been used to treat approximately 7,000 PKU patients around the world. The company has two approved PKU therapies, and the investigational gene therapy BMN 307 is currently in development. BioMarin has conducted 41 clinical studies in PKU and has sponsored 44 external clinical studies. BioMarin researchers have authored 65 publications in medical and scientific journals on PKU and supported another 57 publications by external researchers.

About Gene Therapy

Gene therapy is a form of treatment designed to address a genetic problem by adding a normal copy of the defective gene. The functional gene is inserted into a vector containing a small DNA sequence that acts as a delivery mechanism, providing the ability to deliver the functional gene to targeted cells. The cells can then use the information from the normal gene to build the functional proteins that the body needs, potentially reducing or eliminating the cause of the disease.

Gene Therapy Manufacturing

BioMarin has leveraged its knowledge and experience in manufacturing complex biological products to design, construct and validate a state-of-the-art vector production facility in Novato, California. This facility is the site of production for both valoctocogene roxaparvovec and BMN 307, investigational gene therapies. Manufacturing capabilities are an essential driver for BioMarin's gene therapy programs and allows the Company to control quality, capacity, costs and scheduling enabling rapid development. Production of BMN 307 with a commercial ready process at scale reduces risk associated with making process changes later in development and may speed overall development timelines significantly.

Ongoing process development efforts and experience gained at commercial scale have led to improvements in productivity and operational efficiency. The ability to scale out the facility with additional equipment combined with the improvements in productivity result in a doubling of overall potential capacity to 10,000 doses per year, combined for both products, depending on final dose and product mix. This improvement in productivity is anticipated to meet potential commercial and clinical demand for both valoctocogene roxaparvovec and BMN 307 well into the future.

About Phenylketonuria

PKU, or phenylalanine hydroxylase (PAH) deficiency, is a genetic disorder affecting approximately 70,000 diagnosed patients in the regions of the world where BioMarin operates and is caused by a deficiency of the enzyme PAH. This enzyme is required for the metabolism of Phe, an essential amino acid found in most protein-containing foods. If the active enzyme is not present in sufficient quantities, Phe accumulates to abnormally high levels in the blood and becomes toxic to the brain, resulting in a variety of complications including severe intellectual disability, seizures, tremors, behavioral problems and psychiatric symptoms. As a result of newborn screening efforts implemented in the 1960s and early 1970s, virtually all individuals with PKU under the age of 40 in countries with newborn screening programs are diagnosed at birth and treatment is implemented soon after. PKU can be managed with a severe Phe-restricted diet, which is supplemented by low-protein modified foods and Phe-free medical foods; however, it is difficult for most patients to adhere to the life-long strict diet to the extent needed to achieve adequate control of blood Phe levels. Dietary control of Phe in childhood can prevent major developmental neurological toxicities, but poor control of Phe in adolescence and adulthood is associated with a range of neurocognitive disabilities with significant functional impact.

To learn more about PKU and PAH deficiency, please visit http://www.PKU.com. Information on this website is not incorporated by reference into this press release.

About BioMarin

BioMarin is a global biotechnology company that develops and commercializes innovative therapies for patients with serious and life-threatening rare and ultra-rare genetic diseases.The company's portfolio consists of six commercialized products and multiple clinical and pre-clinical product candidates.For additional information, please visitwww.biomarin.com. Information on such website is not incorporated by reference into this press release.

Forward-Looking Statement

This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including, without limitation, statements about: the Company's BMN 307 program being eligible to apply for Accelerated Approval and Priority Review if relevant criteria are met, as well as Rolling Review, the development of BioMarin's BMN 307 program generally, including the impact on the timing and process for regulatory interactions and decisions, BioMarin's gene therapy manufacturing capabilities and the anticipation that the current manufacturing capabilities will meet potential commercial and clinical demand for both valoctocogene roxaparvovec and BMN 307 well into the future and the impact of using material manufactured at commercial scale in a clinical trial on reducing risk and speeding up overall development timelines. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others:the content and timing of decisions by the U.S. Food and Drug Administration, the European Commission and other regulatory authorities; uncertainties inherent in research and development, including unfavorable new clinical data and additional analyses of existing clinical data; the results and timing of current and future clinical trials related to BMN 307; our ability to reproducibly and consistently manufacture sufficient quantities of BMN 307, the possibility that changes may be required to the current manufacturing process; and those factors detailed in BioMarin's filings with the Securities and Exchange Commission (SEC), including, without limitation, the factors contained under the caption "Risk Factors" in BioMarin's Quarterly Report on Form 10-Q for the quarter ended June 30, 2020 as such factors may be updated by any subsequent reports. Stockholders are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise.

BioMarin is a registered trademark of BioMarin Pharmaceutical Inc.

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BioMarin, Pioneer in Phenylketonuria (PKU) and Gene Therapy, Receives FDA Fast Track Designation for PKU Investigational Gene Therapy, BMN 307 -...

Cell and Gene Therapy Market research report is the new statistical data source added by A2Z Market Research.

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Market Segmentation by Type:

Cell TherapyGene Therapy

Market Segmentation by Application:

HospitalsWound Care CentersCancer Care CentersAmbulatory Surgical CentersOthers

Regions Covered in the Global Cell and Gene Therapy Market Report 2020:The Middle East and Africa(GCC Countries and Egypt)North America(the United States, Mexico, and Canada)South America(Brazil etc.)Europe(Turkey, Germany, Russia UK, Italy, France, etc.)Asia-Pacific(Vietnam, China, Malaysia, Japan, Philippines, Korea, Thailand, India, Indonesia, and Australia)

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Table of Contents

Global Cell and Gene Therapy Market Research Report 2020 2026

Chapter 1 Cell and Gene Therapy Market Overview

Chapter 2 Global Economic Impact on Industry

Chapter 3 Global Market Competition by Manufacturers

Chapter 4 Global Production, Revenue (Value) by Region

Chapter 5 Global Supply (Production), Consumption, Export, Import by Regions

Chapter 6 Global Production, Revenue (Value), Price Trend by Type

Chapter 7 Global Market Analysis by Application

Chapter 8 Manufacturing Cost Analysis

Chapter 9 Industrial Chain, Sourcing Strategy and Downstream Buyers

Chapter 10 Marketing Strategy Analysis, Distributors/Traders

Chapter 11 Market Effect Factors Analysis

Chapter 12 Global Cell and Gene Therapy Market Forecast

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Comprehensive Report on Cell and Gene Therapy Market 2020 | Trends, Growth Demand, Opportunities & Forecast To 2026 | JW CreaGene, Vericel, Tego...

NEW YORK--(BUSINESS WIRE)--Pfizer Inc. (NYSE: PFE) today announced that its investigational gene therapy candidate (PF-06939926) being developed to treat Duchenne muscular dystrophy (DMD) received Fast Track designation from the U.S. Food and Drug Administration (FDA). PF-06939926 is currently being evaluated to determine the safety and efficacy of this gene therapy in boys with DMD.

Fast Track is a process designed to facilitate the development, and expedite the review, of new drugs that are intended to treat or prevent serious conditions that have the potential to address an unmet medical need. This designation was granted based on data from the Phase 1b study that indicated that the intravenous administration of PF-06939926 was well-tolerated during the infusion period and dystrophin expression levels were sustained over a 12-month period.

The FDAs decision to grant our investigational gene therapy PF-06939926 Fast Track designation underscores the urgency to address a significant unmet treatment need for Duchenne muscular dystrophy, said Brenda Cooperstone, MD, Chief Development Officer, Rare Disease, Pfizer Global Product Development. DMD is a devasting condition and patients, and their parents, are waiting desperately for treatment options. We are working to advance our planned Phase 3 program as quickly as possible.

DMD is a devastating and life-threatening X-linked disease that is caused by mutations in the gene encoding dystrophin, which is needed for proper muscle membrane stability and function. Patients present with muscle degeneration that progressively worsens with age to the extent that they require wheelchair assistance when they are in their early teens, and unfortunately, usually succumb to their disease by the time they are in their late twenties. It is estimated that there are ~10-12,000 individuals affected with DMD in the US.

About PF-06939926

PF-06939926 is an investigational, recombinant adeno-associated virus serotype 9 (rAAV9) capsid carrying a shortened version of the human dystrophin gene (mini-dystrophin) under the control of a human muscle-specific promotor. The rAAV9 capsid was chosen as the delivery vector because of its potential to target muscle tissue. Pfizer initiated the Phase 1b multi-center, open-label, non-randomized, ascending dose study of a single intravenous infusion of PF-06939926 in 2018. The goal of the study is to assess the safety and tolerability of this investigational gene therapy. Other objectives of the clinical study include measurement of dystrophin expression and distribution, as well as assessments of muscle strength, quality and function.

About Pfizer Rare Disease

Rare disease includes some of the most serious of all illnesses and impacts millions of patients worldwide, representing an opportunity to apply our knowledge and expertise to help make a significant impact on addressing unmet medical needs. The Pfizer focus on rare disease builds on more than two decades of experience, a dedicated research unit focusing on rare disease, and a global portfolio of multiple medicines within a number of disease areas of focus, including rare hematologic, neurologic, cardiac and inherited metabolic disorders.

Pfizer Rare Disease combines pioneering science and deep understanding of how diseases work with insights from innovative strategic collaborations with academic researchers, patients, and other companies to deliver transformative treatments and solutions. We innovate every day leveraging our global footprint to accelerate the development and delivery of groundbreaking medicines and the hope of cures.

Click here to learn more about our Rare Disease portfolio and how we empower patients, engage communities in our clinical development programs, and support programs that heighten disease awareness.

Pfizer Inc.: Breakthroughs that change patients lives

At Pfizer, we apply science and our global resources to bring therapies to people that extend and significantly improve their lives. We strive to set the standard for quality, safety and value in the discovery, development and manufacture of health care products, including innovative medicines and vaccines. Every day, Pfizer colleagues work across developed and emerging markets to advance wellness, prevention, treatments and cures that challenge the most feared diseases of our time. Consistent with our responsibility as one of the world's premier innovative biopharmaceutical companies, we collaborate with health care providers, governments and local communities to support and expand access to reliable, affordable health care around the world. For more than 150 years, we have worked to make a difference for all who rely on us. We routinely post information that may be important to investors on our website at http://www.pfizer.com. In addition, to learn more, please visit us on http://www.pfizer.com and follow us on Twitter at @Pfizer and @Pfizer_News, LinkedIn, YouTube and like us on Facebook at Facebook.com/Pfizer.

DISCLOSURE NOTICE: The information contained in this release is as of October 1, 2020. Pfizer assumes no obligation to update forward-looking statements contained in this release as the result of new information or future events or developments.

This release contains forward-looking information about PF-06939926, an investigational gene therapy to potentially treat Duchenne muscular dystrophy, including its potential benefits and a planned Phase 3 study for PF-06939926, that involve substantial risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statements. Risks and uncertainties include, among other things, the uncertainties inherent in research and development, including the ability to meet anticipated clinical endpoints, commencement and/or completion dates for our clinical trials, regulatory submission dates, regulatory approval dates and/or launch dates, as well as the possibility of unfavorable new clinical data and further analyses of existing clinical data; the risks associated with initial and preliminary data; the risk that clinical trial data are subject to differing interpretations and assessments by regulatory authorities; whether regulatory authorities will be satisfied with the design of and results from our clinical studies; whether and when regulatory authorities will approve the commencement of our planned Phase 3 study; whether and when drug applications may be filed in any jurisdictions for any potential indication for PF-06939926; whether and when any such applications may be approved by regulatory authorities, which will depend on myriad factors, including making a determination as to whether the product's benefits outweigh its known risks and determination of the product's efficacy and, if approved, whether PF-06939926 will be commercially successful; decisions by regulatory authorities impacting labeling, manufacturing processes, safety and/or other matters that could affect the availability or commercial potential of PF-06939926; uncertainties regarding the impact of COVID-19 on our business, operations and financial results; and competitive developments.

A further description of risks and uncertainties can be found in Pfizers Annual Report on Form 10-K for the fiscal year ended December 31, 2019 and in its subsequent reports on Form 10-Q, including in the sections thereof captioned Risk Factors and Forward-Looking Information and Factors That May Affect Future Results, as well as in its subsequent reports on Form 8-K, all of which are filed with the U.S. Securities and Exchange Commission and available at http://www.sec.gov and http://www.pfizer.com.

Read more:
Pfizer Receives FDA Fast Track Designation for Duchenne Muscular Dystrophy Investigational Gene Therapy - Business Wire

Latest Research Report: Gene Therapy industry

Gene Therapy Market report is to provide accurate and strategic analysis of the Profile Projectors industry. The report closely examines each segment and its sub-segment futures before looking at the 360-degree view of the market mentioned above. Market forecasts will provide deep insight into industry parameters by accessing growth, consumption, upcoming market trends and various price fluctuations.

This has brought along several changes in This report also covers the impact of COVID-19 on the global market.

Gene Therapy Market competition by top manufacturers as follow:SangamoSpark TherapeuticsDimension TherapeuticsAvalanche BioCelladonVicalAdvantagene

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Global Gene Therapy Market research reports growth rates and market value based on market dynamics, growth factors. Complete knowledge is based on the latest innovations in the industry, opportunities and trends. In addition to SWOT analysis by key suppliers, the report contains a comprehensive market analysis and major players landscape.The Type Coverage in the Market are: Ex vivoIn vivo

Market Segment by Applications, covers:Cancer DiseasesMonogenic DiseasesInfectious DiseasesCardiovascular DiseasesOthers

Market segment by Regions/Countries, this report coversNorth AmericaEuropeChinaRest of Asia PacificCentral & South AmericaMiddle East & Africa

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Gene Therapy Market Incredible Possibilities, Growth Analysis and Forecast To 2025 - The Daily Chronicle

The pharmaceutical industry is one of the most scientifically innovative and competitive industries, particularly in oncology. As of 2018, there were over 1,100 cancer therapies in development, and as of 2020, 362 of them were cell and gene therapies. As a result, there is a need for continued innovation and increased efficiency in terms of drug development to manage cost, complexity and speed to provide potentially transformative therapies for cancer patients.

Within the last two decades, large pharmaceutical corporations have established themselves firmly in oncology by prioritising internal R&D efforts, as well as developing and accessing novel science and technology through collaborations and alliances with biotech companies and academic institutions.

Dramatic advances in the understanding of basic molecular mechanisms of underlying disease has continued to shift R&D focus toward precision medicine choosing the right therapy for a patient based on molecular understanding of their disease and less on traditional cancer therapies such as cytotoxic chemotherapies and broad-cell cycle inhibitors.

As a result of this shift in drug development, a highly concentrated overlay in product modalities and mechanisms of action has crowded the oncology pipeline across a very broad range of hematological and solid tumour indications.

The industry is asking itself how to stay innovative, how to develop and bring to market higher quality therapies to patients and how to do this faster and more efficiently.

A diversity of collaboration types

There is broad recognition that given the breadth and complexity of emerging science driving innovation in oncology, collaborations are essential in order that relevant expertise, know-how and capabilities can be combined in the right way to address patient needs.

Such collaborations take on many forms, ranging from early, multi-party alliances and consortia which are often pre-competitive in nature driving the development and shared learnings from technologies that may be enabling the field as a whole, through to more bespoke collaborations between entities.

Cell therapy research has been built on collaborations amongst scientists and entrepreneurs, providing early proof of concept for modalities thought to be too difficult to commercialise but with a strong potential for patient benefit

These may be more focused on collaborative research and development of novel products, to secure the necessary data for regulatory approvals to make such products available widely to the patients who can benefit from them.

Pre-competitive collaborations, often in basic and preclinical research, can reduce the barrier of competition and drive benefits for all stakeholders, most notably, the patient. As summarised by The National Institutes of Health, this includes reducing the number of redundant clinical trials, enhancing the statistical strength of studies, reducing overall costs and risks, and improving study participant recruitment, all while triggering creativity and innovation between collaborators.

These benefits strengthen capabilities and accelerate product development, ultimately producing higher quality and more effective therapies.

One powerful example is The National Institutes of Healths Partnership for Accelerating Cancer Therapies (PACT), which brought together 11 pharmaceutical companies to accelerate the development of new cancer immunotherapies.

Aligning with the focus of the Cancer Moonshot Research Initiative, PACT aimed to retrospectively analyse patient data from past clinical trials with the goal of predicting future patient outcomes.

This type of approach supports the ability to compare data across all trials and facilitates information sharing between partners, undoubtedly accelerating the pathway to effective therapies.

A second example is the establishment of The Parker Institute for Cancer Immunotherapy, to enable leading academic researchers and companies to come together in a pre-competitive setting, to enable rapid shared understanding and development of immunotherapeutic approaches, including the study of combination regimens.

Such combination trials, particularly those encompassing investigational products, have historically been challenging to undertake given the need for bespoke company-to-company and other 1:1 collaborative agreements. Bringing together multiple academic and industry participants under an open innovation model provides a basis to significantly accelerate the generation of scientific and clinical data that may substantially inform the field of cancer immunotherapy as a whole.

Oncology cell therapy research has been built on foundational academic collaborations amongst scientists and entrepreneurs, providing early proof of concept for modalities thought to be too difficult to commercialise but with a strong potential for patient benefit.

Examples include Kite Pharma, formed from the foundational work at the National Cancer Institute, Juno from the collaboration between the Fred Hutchinson Cancer Center and Memorial Sloan Kettering Cancer Center (all working on the first CAR T-cell candidates), or Adaptimmune working with University of Penn to first show efficacy of optimised TCR T-cells.

For collaborations that are more geared to development of novel therapies, aiming for regulatory approval and commercial availability, bespoke collaborations between biotech and pharma companies are commonplace, whereby the respective expertise and capabilities of each partner are combined in order to optimise and accelerate development, and to enable subsequent, larger scale manufacture and distribution. There are many examples of such collaborations, for which the structure can vary widely depending on the expertise of each partner, and the collaborative ways of working.

For example, under a traditional pharma/biotech collaboration and licensing model, a biotech partner may have primary responsibility for significant elements of research and early product development, and the pharma partner may lead the majority of later stage development, as well as post-approval commercial manufacture and supply. This logically aligns with organisational expertise and scale, and this type of collaboration structure has historically proven to work well. Many novel therapies have been successfully developed through such partnerships.

The rapid emergence of cell and gene therapy has required the industry to establish new and distinct capabilities, such as optimal process development and manufacture of autologous, patient specific cell therapies, whilst minimising the vein-to-vein time (the elapsed time between apheresis treatment for a patient, and reinfusing the final autologous manufactured product).

There are a growing number of biotech and pharma companies that have established or are establishing such end-to-end cell therapy capabilities, which can also play into how collaborations are structured in the field.

Case Study: From Technology Agreement to co-development and co-commercialisation partnership

In 2015, Adaptimmune and Universal Cells signed an agreement to drive the development of technologies leveraging gene-edited Induced Pluripotent Stem Cell (iPSC) lines, towards the development of allogeneic, or off-the-shelf, T-cell therapies. Universal Cells brought leading gene editing capability to make targeted gene edits to modify the characteristics of selected iPSC cell lines, and Adaptimmune the technology to differentiate iPSCs into T-cells.

Back then the science for this collaboration was early and under-developed with both parties embarking on a long-term effort and making significant at-risk investments to determine if edited, functional T-cells could be produced.

Today, Universal Cells (now an Astellas company) and Adaptimmune have established capabilities and expertise to progress novel cell therapies into clinical development, as well as with manufacturing and supply chain.

Based on this progress, in January 2020, Adaptimmune and Astellas signed a product-focused agreement to co-develop and co-commercialise up to three new stem-cell derived allogeneic T-cell therapies for people with cancer.

Given the scientific synergy between Universal Cells and Adaptimmune, and that each company is developing capabilities that may effectively address later stage product development and post-approval commercial supply, the 2020 partnership was structured as a co-development and co-commercialisation agreement. It enables the companies to work closely together, throughout the continuum of research, development and commercialisation.

Astellas and Adaptimmune will collaborate through to the end of phase 1, with Universal Cells leading gene editing activities and Adaptimmune leading iPSC to T-cell differentiation, early product characterisation and development. Beyond that, Astellas and Adaptimmune will decide whether to develop and commercialise a product candidate together under a co-development and co-commercialisation cost and profit-sharing arrangement, or for one company to take it forward alone.

This partnership is an example of how companies can harness their individual science and bring together highly complementary skills and expertise. It will enable the development of new, off-the-shelf T-cell therapies for people with cancer, which could potentially offer significant advantages such as broader access, reduced vein-to-vein time, and lower cost. The co-development and co-commercialisation nature of the agreement allows both companies to collaborate closely and on a long term basis, whilst leveraging end-to-end capabilities established by each company, maximising the velocity of product development, and ultimately delivering novel therapies to patients.

This type of agreement exemplifies how early speculative scientific collaboration can benefit all parties, most importantly the patient. It is one example from many in oncology, that underlines the value of long-term partnership within a field that is evolving rapidly across many scientific, operational and commercial frontiers.

Bringing together both teams of passionate and forward-thinking scientists may contribute to unlocking the current opportunities and challenges of off-the-shelf T-cell therapy development more effectively and efficiently for patients.

Similarly to what we are seeing as the world comes together to fight COVID-19, we as leaders in oncology owe it to patients to constantly look for ways to bring our innovative ideas as quickly as possible to the market. Working together might make that happen faster.

About the author

Helen Tayton-Martin is chief business officer at Adaptimmune.

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The new pharma collaborations driving transformative research in oncology - - pharmaphorum

As the COVID-19 pandemic has progressed, we have seen an alarming amount of disinformation spread online, including by our elected officials. In Colorado we have seen elected officials visiting restaurants in the middle of stay-at-home orders without wearing a mask.

Other elected officials have flouted local health department advice by gathering with thousands at Bandimere Speedway, where few wore masks.

We have heard them say that statewide mask mandates somehow infringe on their rights, ignoring that placing the health of others infringes on the rights of so many innocent Coloradans.They have even sued the state government for its attempts to protect public health.

Despite advice from our public health leaders to practice social distancing and wear masks to prevent the spread of the virus, President Donald Trump and Vice President Mike Pence continue to refuse to wear masks in public.

Pence leads the White Houses coronavirus task force, yet he toured the Mayo Clinic without a mask on April 28 (that same day, the U.S. reached one million COVID-19 cases).

The latest from the coronavirus outbreak in Colorado:

>> FULL COVERAGE

Recently, we have learned the alarming fact that President Trump knew back in February that this virus is spread through the air and five times deadlier than the flu.

Amid lies and conspiracy theories, our light at the end of the tunnel is scientific innovation and trust in experts. Thanks to round-the-clock collaboration between the public and private sectors, there are several COVID-19 vaccines and treatments in development.

One, for example, blocks the novel coronavirus from binding to human cells and reproducing; by stopping the virus from connecting with human cells, the drug prevents it from multiplying and attacking the body.

READ:Colorado Sun opinion columnists.

There are more than 100 different vaccines at various stages of development, and researchers are using different avenues such as gene therapy, DNA and antibodies from survivors to develop an effective vaccine.

U.S. health care innovation has saved millions of lives.HIV is now a manageable disease, no longer a death sentence.Thanks to developments in early-detection mammogram technology, female breast cancer cases dropped by 40% in 2016.

We now have a drug that can treat over 90% of Hepatitis C patients, whereas older drugs took nearly a year to become effective and even then only worked on 50% of patients.

And vaccines have saved the health of millions by preventing once-fatal illnesses. History teaches us that our best bet is to support the researchers working to develop treatments and vaccines for COVID-19.

We owe it to the frontline essential workers our grocery store workers, health care workers, sanitation services, public transit operators and so many more risking their lives every day to do better in this pandemic.

We need to ignore disinformation, whether it comes from the internet or the White House and follow the advice of our public health professionals.

We need more medical professionals and scientists to run for office to be that expert voice to help stop the spread of misinformation before it starts. Supporting and investing in their research and innovation will get us through this crisis.

Dr. Yadira Caraveo, D-Thornton, is a pediatrician and represents House District 31 in the Colorado House of Representatives.

The Colorado Sun is a nonpartisan news organization, and the opinions of columnists and editorial writers do not reflect the opinions of the newsroom. Read our ethics policy for more on The Suns opinion policy and submit columns, suggested writers and more to opinion@coloradosun.com.

Support local journalism around the state.Become a member of The Colorado Sun today!

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Opinion: Scientific innovation and trust in experts is our light at the end of the tunnel - The Colorado Sun

NEW YORK--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (Rocket), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces two presentations at the European Society for Immunodeficiencies (ESID) 2020 Meeting to be held virtually October 14-17, 2020. An oral presentation will provide an update on data from the Phase 1/2 clinical trial of RP-L201 for Leukocyte Adhesion Deficiency-I (LAD-I). An e-poster will highlight preclinical study data on RP-L401 for Infantile Malignant Osteopetrosis (IMO).

Additional presentation details can be found below:

Oral Presentation

Title: A Phase 1/2 Study of Lentiviral-Mediated Ex-Vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I): Results from Phase 1 Session Title: TreatmentPresenter: Donald B. Kohn, M.D., Professor of Microbiology, Immunology and Molecular Genetics, Pediatrics (Hematology/Oncology), Molecular and Medical Pharmacology, and member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at the University of California, Los AngelesSession Date: Friday, October 16, 2020Session Time: 10:45 a.m. 12:01 p.m. CESTLecture Time: 11:45 a.m. CESTLocation: Hall D

This session will be followed by a Q&A from 12:01 p.m. to 12:30 p.m. CEST

E-Poster

Title: Preclinical Efficacy and Safety of EFS.HTCIRG1-LV Supports IMO Gene Therapy Clinical Trial InitiationPresenter: Ilana Moscatelli, Ph.D., Associate Researcher, Division of Molecular Medicine and Gene Therapy, Lund University, Sweden

About Leukocyte Adhesion Deficiency-I

Severe Leukocyte Adhesion Deficiency-I (LAD-I) is a rare, autosomal recessive pediatric disease caused by mutations in the ITGB2 gene encoding for the beta-2 integrin component CD18. CD18 is a key protein that facilitates leukocyte adhesion and extravasation from blood vessels to combat infections. As a result, children with severe LAD-I (less than 2% normal expression) are often affected immediately after birth. During infancy, they suffer from recurrent life-threatening bacterial and fungal infections that respond poorly to antibiotics and require frequent hospitalizations. Children who survive infancy experience recurrent severe infections including pneumonia, gingival ulcers, necrotic skin ulcers, and septicemia. Without a successful bone marrow transplant, mortality in patients with severe LAD-I is 60-75% prior to the age of 2 and survival beyond the age of 5 is uncommon. There is a high unmet medical need for patients with severe LAD-I.

Rockets LAD-I research is made possible by a grant from the California Institute for Regenerative Medicine (Grant Number CLIN2-11480). The contents of this press release are solely the responsibility of Rocket and do not necessarily represent the official views of CIRM or any other Agency of the State of California.

About Infantile Malignant Osteopetrosis

Infantile Malignant Osteopetrosis (IMO) is a rare, severe autosomal recessive disorder caused by mutations in the TCIRG1 gene, which is critical for the process of bone resorption. Mutations in TCIRG1 interfere with the function of osteoclasts, cells which are essential for normal bone remodeling and growth, leading to skeletal malformations, including fractures and cranial deformities which cause neurologic abnormalities including vision and hearing loss. Patients often have endocrine abnormalities and progressive, frequently fatal bone marrow failure. As a result, death is common within the first decade of life. IMO has an estimated incidence of 1 in 200,000. The only treatment option currently available for IMO is an allogenic bone marrow transplant (HSCT), which allows for the restoration of bone resorption by donor-derived osteoclasts which originate from hematopoietic cells. Long-term survival rates are lower in IMO than those associated with HSCT for many other non-malignant hematologic disorders; severe HSCT-related complications are frequent. There is an urgent need for additional treatment options.

RP-L401 was in-licensed from Lund University and Medizinische Hochschule Hannover.

About Rocket Pharmaceuticals, Inc.

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (Rocket) is advancing an integrated and sustainable pipeline of genetic therapies that correct the root cause of complex and rare childhood disorders. The companys platform-agnostic approach enables it to design the best therapy for each indication, creating potentially transformative options for patients afflicted with rare genetic diseases. Rocket's clinical programs using lentiviral vector (LVV)-based gene therapy are for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer, Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, Pyruvate Kinase Deficiency (PKD) a rare, monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia and Infantile Malignant Osteopetrosis (IMO), a bone marrow-derived disorder. Rockets first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon disease, a devastating, pediatric heart failure condition. For more information about Rocket, please visit http://www.rocketpharma.com.

Rocket Cautionary Statement Regarding Forward-Looking Statements

Various statements in this release concerning Rocket's future expectations, plans and prospects, including without limitation, Rocket's expectations regarding its guidance for 2020 in light of COVID-19, the safety, effectiveness and timing of product candidates that Rocket may develop, to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), Infantile Malignant Osteopetrosis (IMO) and Danon Disease, and the safety, effectiveness and timing of related pre-clinical studies and clinical trials, may constitute forward-looking statements for the purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995 and other federal securities laws and are subject to substantial risks, uncertainties and assumptions. You should not place reliance on these forward-looking statements, which often include words such as "believe," "expect," "anticipate," "intend," "plan," "will give," "estimate," "seek," "will," "may," "suggest" or similar terms, variations of such terms or the negative of those terms. Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, Rocket's ability to monitor the impact of COVID-19 on its business operations and take steps to ensure the safety of patients, families and employees, the interest from patients and families for participation in each of Rockets ongoing trials, our expectations regarding when clinical trial sites will resume normal business operations, our expectations regarding the delays and impact of COVID-19 on clinical sites, patient enrollment, trial timelines and data readouts, our expectations regarding our drug supply for our ongoing and anticipated trials, actions of regulatory agencies, which may affect the initiation, timing and progress of pre-clinical studies and clinical trials of its product candidates, Rocket's dependence on third parties for development, manufacture, marketing, sales and distribution of product candidates, the outcome of litigation, and unexpected expenditures, as well as those risks more fully discussed in the section entitled "Risk Factors" in Rocket's Annual Report on Form 10-Q for the quarter ended June 30, 2020, filed August 5, 2020 with the SEC. Accordingly, you should not place undue reliance on these forward-looking statements. All such statements speak only as of the date made, and Rocket undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

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Rocket Pharmaceuticals Announces Two Presentations at the European Society for Immunodeficiencies 2020 Meeting - Business Wire

LONDONandNEW YORK, Oct. 03, 2020 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc(Nasdaq: MGTX), a vertically integrated, clinical stage gene therapy company, today announced nine-month data from the ongoing Phase 1/2 clinical trial (NCT03252847) of AAV-RPGR, an investigational gene therapy in development for the treatment of patients with X-linked retinitis pigmentosa (XLRP). Data presented today at the EURETINA 2020 Virtual Congress demonstrated significant improvement in vision-guided mobility and retinal sensitivity in treated eyes compared to untreated eyes nine months after treatment.

MeiraGTx and Janssen Pharmaceuticals, Inc. (Janssen), one of the Janssen Pharmaceutical Companies of Johnson & Johnson, are jointly developing AAV-RPGR as part of a broader collaboration to develop and commercialize gene therapies for the treatment of inherited retinal diseases.

In July 2020, MeiraGTx and Janssen announced six-month data from the ongoing MGT009 clinical trial showing significant improvement in retinal sensitivity in the low (n=3) and intermediate (n=4) dose cohorts in the dose escalation phase of the trial.

Data at the nine-month time point continued to demonstrate significant improvement in retinal sensitivity in treated eyes in both the low and intermediate dose cohorts. In addition, data from the assessment of vision-guided mobility carried out at the nine-month timepoint demonstrated a significant improvement in walk time compared to baseline in treated eyes compared to untreated eyes in the low and intermediate dose cohorts (n=6).

There are currently no treatment options for XLRP, and vision in patients suffering from this disease inevitably declines over time, said Michel Michaelides1, BSc MB BS MD(Res) FRCOphth FACS, MGT009 trial investigator, Consultant Ophthalmologist, Moorfields Eye Hospital and Professor of Ophthalmology, University College London. Data from this clinical trial demonstrate that patients treated with AAV-RPGR had significant and sustained improvement in retinal sensitivity, as well as improved ability to navigate in low light conditions. These exciting results continue to suggest that AAV-RPGR has the potential to be a much-needed and important treatment option for those living with XLRP.

EURETINA Data Summary:

Retinal sensitivity XLRP is characterized by progressive deterioration of the visual field. Octopus 900 full-field static perimetry and MAIA microperimetry were employed to determine change in retinal sensitivity following intervention.

Perimetry is a sensitive standard-of-care measure of retinal function that reproducibly determines retinal sensitivity both cross-sectionally and longitudinally, thereby accurately defining disease progression over time.

At the nine-month analysis (Octopus 900 static perimetry), compared to baseline:

Vision-guided mobility Markedly impaired mobility in low illumination is a hallmark symptom of XLRP. As part of the study, patients completed a vision-guided mobility maze to assess their ability to navigate across a broad range of controlled light levels (1 lux = deep twilight, 4 lux = residential street lighting, 16 lux = twilight conditions, 64 lux = car park and 256 lux = office work).

At nine-month analysis, compared to baseline:

Safety and tolerabilitySafety data obtained to date continue to suggest AAV-RPGR is well-tolerated. No dose-limiting events occurred. As previously presented, signs of inflammation were observed in two out of three patients in the high dose cohort, which may have been associated with decreased activity of the AAV-RPGR treatment in these patients. Inflammation was effectively managed with an extended steroid protocol.

Based on the safety and efficacy profile demonstrated to date, the low and intermediate doses are being evaluated in the ongoing randomized, controlled expansion portion of the Phase 1/2 study, which completed enrollment in the first half of 2020. As previously disclosed, MeiraGTx and development partner Janssen plan to advance AAV-RPGR into a Phase 3 pivotal study, called the Lumeos clinical trial.

About AAV-RPGRAAV-RPGR is an investigational gene therapy for the treatment of patients with XLRP caused by disease-causing variants in the eye specific form of the RPGR gene (RPGR ORF15). AAV-RPGR is designed to deliver functional copies of the RPGR gene to the subretinal space in order to improve and preserve visual function. MeiraGTx and development partner Janssen are currently conducting a Phase 1/2 clinical trial of AAV-RPGR in patients with XLRP with disease-causing variants in RPGR ORF15. AAV-RPGR has been granted Fast Track and Orphan Drug designations by the U.S. Food and Drug Administration (FDA) and PRIME, ATMP and Orphan designations by the European Medicines Agency (EMA).

About the Phase 1/2 MGT009 Clinical TrialMGT009 is a multi-center, open-label Phase 1/2 trial (NCT03252847) of AAV-RPGR gene therapy for the treatment of patients with XLRP associated with disease-causing variants in the RPGR gene. MGT009 consists of three phases: dose-escalation, dose-confirmation, and dose-expansion. Each patient was treated with subretinal delivery of AAV-RPGR in the eye that was more affected at baseline. The patients other eye served as an untreated control. In dose-escalation (n=10), adults were administered low, intermediate, or high dose AAV-RPGR. The primary endpoint was safety. Visual function was assessed at baseline, three, six, nine and 12 months with Octopus 900 full-field static perimetry and mesopic fundus-guided microperimetry (MP); mean retinal sensitivity, visual field modeling and analysis (VFMA; Hill-of-vision volumetric measure), and pointwise comparisons were examined.

About X-Linked Retinitis Pigmentosa (XLRP)XLRP is the most severe form of retinitis pigmentosa (RP), a group of inherited retinal diseases characterized by progressive retinal degeneration and vision loss. In XLRP, both rods and cones function poorly, leading to degeneration of the retina and total blindness. The most frequent cause of XLRP is disease-causing variants in the RPGR gene, accounting for more than 70% of cases of XLRP, and up to 20% of all cases of RP. There are currently no approved treatments for XLRP.

AboutMeiraGTxMeiraGTx(Nasdaq: MGTX) is a vertically integrated, clinical stage gene therapy company with six programs in clinical development and a broad pipeline of preclinical and research programs.MeiraGTx has core capabilities in viral vector design and optimization and gene therapy manufacturing, as well as a potentially transformative gene regulation technology. Led by an experienced management team,MeiraGTxhas taken a portfolio approach by licensing, acquiring and developing technologies that give depth across both product candidates and indications. MeiraGTxs initial focus is on three distinct areas of unmet medical need: inherited retinal diseases, neurodegenerative diseases and severe forms of xerostomia. Though initially focusing on the eye, central nervous system and salivary gland,MeiraGTxintends to expand its focus in the future to develop additional gene therapy treatments for patients suffering from a range of serious diseases.

For more information, please visit http://www.meiragtx.com.

Forward Looking StatementThis press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including, without limitation, statements regarding the development and efficacy of AAV-RPGR, plans to advance AAV-RPGR into Phase 3 clinical trial and anticipated milestones regarding our clinical data and reporting of such data and the timing of results of data, including in light of the COVID-19 pandemic, as well as statements that include the words expect, intend, plan, believe, project, forecast, estimate, may, should, anticipate and similar statements of a future or forward-looking nature. These forward-looking statements are based on managements current expectations. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, our incurrence of significant losses; any inability to achieve or maintain profitability, acquire additional capital, identify additional and develop existing product candidates, successfully execute strategic priorities, bring product candidates to market, expansion of our manufacturing facilities and processes, successfully enroll patients in and complete clinical trials, accurately predict growth assumptions, recognize benefits of any orphan drug designations, retain key personnel or attract qualified employees, or incur expected levels of operating expenses; the impact of the COVID-19 pandemic on the status, enrollment, timing and results of our clinical trials and on our business, results of operations and financial condition; failure of early data to predict eventual outcomes; failure to obtain FDA or other regulatory approval for product candidates within expected time frames or at all; the novel nature and impact of negative public opinion of gene therapy; failure to comply with ongoing regulatory obligations; contamination or shortage of raw materials or other manufacturing issues; changes in healthcare laws; risks associated with our international operations; significant competition in the pharmaceutical and biotechnology industries; dependence on third parties; risks related to intellectual property; changes in tax policy or treatment; our ability to utilize our loss and tax credit carryforwards; litigation risks; and the other important factors discussed under the caption Risk Factors in our Quarterly Report on Form 10-Q for the quarter ended June 30, 2020, as such factors may be updated from time to time in our other filings with the SEC, which are accessible on the SECs website at http://www.sec.gov. These and other important factors could cause actual results to differ materially from those indicated by the forward-looking statements made in this press release. Any such forward-looking statements represent managements estimates as of the date of this press release. While we may elect to update such forward-looking statements at some point in the future, unless required by law, we disclaim any obligation to do so, even if subsequent events cause our views to change. Thus, one should not assume that our silence over time means that actual events are bearing out as expressed or implied in such forward-looking statements. These forward-looking statements should not be relied upon as representing our views as of any date subsequent to the date of this press release.

Contacts

Investors:MeiraGTxElizabeth (Broder) Anderson(646) 860-7983elizabeth@meiragtx.com

or

Media:W2O pureChristiana Pascale (212) 257-6722cpascale@purecommunications.com

_____________________________1 Professor Michaelides is a scientific founder of and consultant to MeiraGTx.

Originally posted here:
MeiraGTx Announces Nine-Month Data from Phase 1/2 Trial of AAV-RPGR Demonstrating Significant and Sustained Vision Improvement in X-Linked Retinitis...

Gene Therapy Market analysis report encompasses infinite knowledge and information on what the markets definition, classifications, applications, and engagements are and also explains the drivers & restraints of the market which is obtained from SWOT analysis. Gathered market data and information is denoted very neatly with the help of most appropriate graphs, charts or tables in the entire report. Utilization of well established tools and techniques in this Gene Therapy Market document helps to turn complex market insights into simpler version. Competitive analysis studies of this market report provides with the ideas about the strategies of key players in the market.

A large scale Gene Therapy Market report endows with the data and statistics on the current state of the industry which directs companies and investors interested in this market. By applying market intelligence for this market research report, industry expert measure strategic options, summarize successful action plans and support companies with critical bottom-line decisions. The most appropriate, unique, and creditable global market report has been brought to important customers and clients depending upon their specific business needs. Businesses can accomplish great benefits with the different & all-inclusive segments covered in the Gene Therapy Market research report hence every bit of market is tackled carefully.

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Market Analysis: Global Gene Therapy Market

Global gene therapy market is rising gradually with a healthy CAGR of 36.1% in the forecast period of 2019-2026. Increasing incidence of cancer and rare life threatening diseases and strong clinical pipeline drugs for gene therapy are major drivers for market growth.

Key Market Players:

Few of the major competitors currently working in the globalgene therapy marketarePfizer Inc., Thermo Fisher Scientific Inc., F. Hoffmann-La Roche Ltd, Spark Therapeutics, Inc., bluebird bio, Inc., ALLERGAN, Krystal Biotech, Inc., Amicus Therapeutics, Inc., Sarepta Therapeutics, Novartis AG, MeiraGTx Limited, Rocket Pharmaceuticals, Lonza, Biogen, Gilead Sciences, Inc., REGENXBIO Inc., uniQure N.V., Solid Biosciences Inc., Audentes Therapeutics among others.

Get Full TOC, Tables and Figures of Market Report @https://www.databridgemarketresearch.com/toc/?dbmr=global-gene-therapy-market&rp

Market Definition: Global Gene Therapy Market

Gene therapy is a technique of insertion of genes into cells and tissues for treatment of any disease. In this technique the defective gene is replaced with a functional gene. It is the strategy of manipulation of expression of specific genes responsible for the disease. This therapy is a promising treatment option for a number of diseases. The application of gene therapy is wide and it is mostly used for treatment of cancer, cystic fibrosis, heart disease, diabetes, AIDS among others.

Gene Therapy Market Drivers

Gene Therapy Market Restraints

Segmentation:Global Gene Therapy Market

Gene Therapy Market : By Type

Gene Therapy Market : By Gene Type

Gene Therapy Market : By Viral Vector

Gene Therapy Market : By Non-Viral Vector

Gene Therapy Market : By Application

Gene Therapy Market : By End Users

Gene Therapy Market : By Distribution Channels

Gene Therapy Market : ByGeography

Key Developments in the Market:

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Gene Therapy Market Sees a Faster Rebound in the Era of COVID-19 || Leading Players ALLERGAN, Krystal Biotech, Inc., Amicus Therapeutics, Inc.,...

Denise Paglinawan , The Canadian Press Published Wednesday, September 30, 2020 10:22PM EDT

The father of a young Ontario boy with a rare genetic disease is asking the federal government to help fund research that could treat his child's condition, saying the support is particularly needed since the COVID-19 pandemic has made it tough to raise donations.

Terry Pirovolakis said his two-year-old son Michael was diagnosed last year with SPG50, an extremely rare disorder that causes a loss of mobility and a decline in brain functions over time.

His family has been raising money for research that could help develop treatment for Michael but the pandemic has meant many planned fundraising events had to be cancelled, he said.

It's been very difficult because we had to stop all of our fundraising campaigns, Pirovolakis said. We're all in the same situation where our lives are turned upside down but ours is a bit more complex, where we're trying to build a cure.

The family noticed Michael was not meeting his milestones as a baby, Pirovolakis said. After many tests and doctors appointments, the young boy was diagnosed with the disease that could lead to developmental delays and confine him to a wheelchair by the age of 10, the family said.

Determined to help their son, the Pirovolakis family has been trying to raise money to get the child into experimental treatment.

The family had put on events that included a golf tournament, a gala and a Christmas market to fundraise last year, but can now only rely largely on online campaigns such as their GoFundMe page while COVID-19 restrictions are still in place, Pirovolakis said.

In an effort to keep raising funds and awareness for their son's condition, Pirovolakis said he planned to bike from Pickering, Ont., to Ottawa - starting on Saturday - and has asked to meet with the Prime Minister Justin Trudeau next week.

We're seeing if there's any way that we can get some sort of funding from the federal government, he said.

Hopefully, we'll meet Mr. Trudeau and bring awareness and bring funding and support not just for my son, but for the other children affected by this disease.

The family has raised almost $1.6 million so far but is hoping to raise $3 million. Pirovolakis said research on SPG50 is being conducted in several hospitals outside Canada and involves gene therapy.

Alex Wellstead, a spokesman for the prime minister's office, said the government is in contact with the family.

It is truly inspiring to see the love and dedication that the Pirovolaskis' have for their son Michael and the hard work they've put towards this campaign, he said in an email. We will let you know when we have more to say.

This report by The Canadian Press was first published on Sept. 30, 2020.

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Family of boy with rare disease asking Ottawa to fund research into condition - CP24 Toronto's Breaking News

Global Gene Therapy Market Research Report 2020: COVID-19 Outbreak Impact Analysis

Brand Essence Market Research has developed a concise study on the Gene Therapy market to depict valuable insights related to significant market trends driving the industry. The report features analysis based on key opportunities and challenges confronted by market leaders while highlighting their competitive setting and corporate strategies for the estimated timeline.

Download Sample PDF (including full TOC, Tables, and Figures) of Gene Therapy Market Research 2020-2026:- https://brandessenceresearch.biz/Request/Sample?ResearchPostId=72699&RequestType=Sample

Global Gene therapy market is valued at USD 0.67 Billion in 2018 and expected to reach USD 5.18 Billion by 2025 with the CAGR of 33.9 % over the forecast period. Increasing prevalence of the cardiovascular disease, cancer, genetic disorder, and painful existing treatment procedure is further driving Gene therapy market.

Gene Therapy is associated with the experimental technique that uses genes to treat or prevent disease. In the future, this system could permit doctors to treat a condition by inserting a gene into patients cells rather than exploitation medication or surgery. Gene therapy replaces a faulty gene or adds a new gene in an attempt to cure disease or improve bodys ability to fight disease. Gene therapy holds potential for treating a wide range of diseases, such as cancer, cystic fibrosis, heart disease, diabetes, hemophilia and AIDS. For example, suppose a brain tumor is forming by rapidly isolating cancer cells.

Global gene therapy market report is segmented on the basis of Therapy type, Vector Type, application, and regional& country level. Based upon Therapy type, gene therapy market is classified as somatic and germline. Based upon Vector type Gene Therapy Market is classified into non-viral vectors and viral vectors. Based upon Application Gene therapy market is classified into Cancer Diseases, Monogenic Diseases, Infectious Diseases, Cardiovascular Diseases, Others.

The regions covered in this Global Gene therapy market report are North America, Europe, Asia-Pacific and Rest of the World. On the basis of country level, market of Gene therapy is sub divided into U.S., Mexico, Canada, UK, France, Germany, Italy, China, Japan, India, South East Asia, GCC, Africa, etc.

Key Players for Global Gene therapy Market ReportsGlobal Gene therapy market Report covers prominent players are like Sangamo, Spark Therapeutics, Dimension Therapeutics, Avalanche Bio, Celladon, Vical, Advantagene.

Increasing prevalence of cardiovascular disease, cancer, genetic disorder, and painful existing treatment procedure is expected to grow Gene therapy market. Growing prevalence of cardiovascular disease, cancer, genetic disorder, and painful existing treatment procedure coupled with the increasing application of public health and clinical facilities is expected to drive Gene therapy market. As this therapy is transforming the current approaches to promote the excellence in health and prevent from cardiovascular diseases such as cancer and diabetes. In 2017, it is projected that around 14% (more than 34 million adults) were current smokers. While People with diabetes comprise 8.8% of the worlds population and IDF predicted that the number of cases of diabetes will rise to 642 million by 2040. In low-income countries, mainly in Africa, the older population faces a considerable burden of both non-communicable and communicable diseases. High cost of gene therapy treatment and unwanted immune responses such factors hindering the growth of gene therapy market. Technological Advancements leading to provide better technological procedures is the lucrative opportunity for gene therapy market in forecast period.

Geographically, this report split global into several key Regions, revenue (Million USD) The geography (North America, Europe, Asia-Pacific, Latin America and Middle East & Africa) focusing on key countries in each region. It also covers market drivers, restraints, opportunities, challenges, and key issues in Global Gene Therapy Market.

Key Benefits for Gene Therapy Market Reports Global market report covers in-depth historical and forecast analysis. Global market research report provides detail information about Market Introduction, Market Summary, Global market Revenue (Revenue USD), Market Drivers, Market Restraints, Market Opportunities, Competitive Analysis, Regional and Country Level. Global market report helps to identify opportunities in market place. Global market report covers extensive analysis of emerging trends and competitive landscape.

By Disease Indication Cancer Genetic disorders Cardiovascular diseases Ophthalmology Neurological conditions Others

By Type of Vectors Viral vectors Non-viral vectors

By Type of Cells Somatic cells Germline cells

By Region North Americao U.S.o Canadao Mexico Europeo UKo Franceo Germanyo Russiao Rest of Europe Asia-Pacifico Chinao South Koreao Indiao Japano Rest of Asia-Pacific LAMEAo Latin Americao Middle Easto Africa

North America is dominating the Gene therapy Market North America is dominating the gene therapy market during the forecast period due to increasing healthcare expenditure coupled with advancement of technologies. The majority of gene therapies clinical trials recognized targeted cancer diseases. In the US, around 66.81% of gene therapy clinical trials are carried out. While all other countries participated in a small percentage of the trials such as 9.45% in the UK, 3.95% in Germany and around 2% each in Switzerland, France, China, and Japan. Globally, regeneration medicine companies providing Gene cell therapy and tissue engineering for therapeutic developers is expected to grow gene therapy market in this region.

Gene Therapy Market Key Players: Pfizer Inc. Novartis AG Bayer AG Sanofi GlaxoSmithKline plc. Amgen Inc. Boehringer Ingelheim International GmbH uniQure N.V. bluebird bio, Inc. Celgene Corporation Others

This comprehensive report will provide:

Enhance your strategic decision making Assist with your research, presentations and business plans Show which emerging market opportunities to focus on Increase your industry knowledge Keep you up-to-date with crucial market developments Allow you to develop informed growth strategies Build your technical insight Illustrate trends to exploit Strengthen your analysis of competitors Provide risk analysis, helping you avoid the pitfalls other companies could make Ultimately, help you to maximize profitability for your company.

Our Market Research Solution Provides You Answer to Below Mentioned Question:

Which are the driving factors responsible for the growth of market? Which are the roadblock factors of this market? What are the new opportunities, by which market will grow in coming years? What are the trends of this market? Which are main factors responsible for new product launch? How big is the global & regional market in terms of revenue, sales and production? How far will the market grow in forecast period in terms of revenue, sales and production? Which region is dominating the global market and what are the market shares of each region in the overall market in 2017? How will each segment grow over the forecast period and how much revenue will these segment account for in 2025? Which region has more opportunities?

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The Essential Content Covered in the GlobalGene Therapy Market Report:

* Top Key Company Profiles.* Main Business and Rival Information* SWOT Analysis and PESTEL Analysis* Production, Sales, Revenue, Price and Gross Margin* Market Share and Size

The report provides a 6-year forecast (2020-2026) assessed based on how the Gene Therapy market is predicted to grow in major regions like USA, Europe, Japan, China, India, Southeast Asia, South America, South Africa, Others.

Strategic Points Covered in TOC:

Chapter 1:Introduction, market driving force product scope, market risk, market overview, and market opportunities of the global Gene Therapy market.

Chapter 2:Evaluating the leading manufacturers of the global Gene Therapy market which consists of its revenue, sales, and price of the products.

Chapter 3: the competitive nature among key manufacturers, with market share, revenue, and sales.

Chapter 4:Presenting global Gene Therapy market by regions, market share and revenue and sales for the projected period.

Chapters 5, 6, 7, 8 and 9:To evaluate the market by segments, by countries and by manufacturers with revenue share and sales by key countries in these various regions.

Read More: https://brandessenceresearch.biz/Lifesciences-and-Healthcare/Gene-Therapy-Market-Share/Summary

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Global Gene Therapy Market 2020: Growth, Latest Trend Analysis and Forecast 2025 - The Daily Chronicle